Educational guides and explanations: learn how each peptide or ingredient works and what the evidence says. This section is our Library: every peptide in common use, graded on the strength of the evidence behind it — A for multiple human trials, D for animal and laboratory work only. How we grade →
AGHK-Cu · copper tripeptide-1
Grade A evidence
Collagen support with human trial evidence behind it.
APalmitoyl peptide blend
Grade A evidence
Around 20% reduction in wrinkle depth at 3% over eight weeks.
BAcetyl hexapeptide-8
Grade B evidence
Genuinely mixed evidence — one independent 2023 evaluation found no measurable effect at all.
BLipidated GHK fragment with better penetration than the parent peptide.
BHydrolysed collagen
Grade B evidence
The one ingestible peptide category with an established lawful status.
AGLP-1 agonist
Grade A evidence
Approved medicine. Proposed for exclusion from the 503B bulks list.
AGIP/GLP-1 agonist
Grade A evidence
Approved medicine. Proposed for exclusion from the 503B bulks list.
BBremelanotide
Grade B evidence
Approved in branded form for a specific indication; widely sold grey-market above label dose.
BKISS1 gene peptide
Grade B evidence
Real human trials exist — for reproductive research, not the libido products it's sold as.
BGHRH(1-29)
Grade B evidence
Genuinely approved once, for a pediatric use — withdrawn in 2008 for commercial reasons, not safety.
CApproved elsewhere for narrow indications; not in the US. Its largest sepsis trial found no benefit.
CLong-acting GHRH analog
Grade C evidence
Real human PD data; FDA's advisory committee voted against it for compounding eligibility.
CGHS-R1a agonist
Grade C evidence
Its one real clinical trial, for bowel recovery, found no benefit over placebo.
DBody protection compound
Grade D evidence
Heavily marketed on almost entirely rodent data. Removed from Category 2 in April 2026 — not the same as permitted.
DThymosin beta-4 fragment
Grade D evidence
The trial evidence cited for it was run on a different, larger molecule. Not a lawful supplement ingredient.
DhGH fragment 176-191
Grade D evidence
A real, dedicated 536-person trial found it doesn't work. Development was halted in 2007.
DEpithalon
Grade D evidence
Longevity claims trace largely to one research group's 2003 cell-culture study.
DMitochondrial ORF peptide
Grade D evidence
Interesting biology; a very wide gap between it and the marketing.
DGHS-R1a agonist
Grade D evidence
Its "cardioprotective" reputation comes from rat hearts, not a human trial.
DGrowth hormone secretagogues
Grade D evidence
GHRP-2 is a real, narrow diagnostic approval in Japan — not the treatment it's sold as.
DNon-selective melanocortin agonist
Grade D evidence
Never approved. Real published case reports link it to melanoma and rhabdomyolysis.
AGLP-1 agonist
Grade A evidence
Approved twice, as Victoza and Saxenda. The GLP-1 that came before Ozempic and Wegovy.
BGIP/GLP-1/glucagon triple agonist
Grade B evidence
Not approved anywhere yet, but real Phase 2 data — and already the target of real FDA warning letters online.
BAmylin analog (± semaglutide)
Grade B evidence
Not yet approved alone or combined — but real Phase 3 data exists and Novo Nordisk reports an FDA application is filed.
BHuman chorionic gonadotropin
Grade B evidence
Really approved for fertility use. FDA's own label says it doesn't work for weight loss.
BSynthetic GnRH
Grade B evidence
Genuinely approved once, for diagnostics and fertility — both now listed as discontinued by FDA.
CACTH(4-10) fragment
Grade C evidence
A registered Russian stroke drug with real, decades-old trial data never replicated in the West.
DDelta sleep-inducing peptide
Grade D evidence
FDA's advisory committee voted it down in 2026 — the only one of seven peptides reviewed to be rejected.
Dα-MSH C-terminal fragment
Grade D evidence
Zero published human trials found, despite a 2026 FDA advisory-committee recommendation for compounding.
CActin-binding protein (full-length)
Grade C evidence
The real protein behind "TB-500." One trial trended positive; a larger 2026 trial missed its endpoint.
CMyostatin/activin inhibitor
Grade C evidence
The only human trial data is from a tiny gene-therapy study — not the injectable peptide sold online.
BA real obstetric drug. Its largest controlled trial for a social/bonding outcome found no benefit.
BProtirelin
Grade B evidence
A real diagnostic drug whose only US product stopped being made in 2002. No trial backs its wellness use.
AA real approved GHRH analog — the one member of this drug class that actually cleared FDA approval.
BSomatropin
Grade B evidence
Genuinely approved for real deficiency states. Prohibited in all sport; no trial evidence for the muscle-building use it's sold for off-label.
ASkytrofa · once-weekly hGH prodrug
Grade A evidence
The same somatropin already used daily, released from an inert carrier over a week instead — approved for children in 2021, adults in 2025.
ASogroya · once-weekly hGH, albumin-binding
Grade A evidence
A different once-weekly engineering trick than lonapegsomatropin — reversible albumin binding, now covering adults plus four pediatric indications.
BAfamelanotide
Grade B evidence
The real, FDA-approved twin of Melanotan II — approved for a rare disease, not the tanning use it's sold for online.
CPorcine brain-derived peptide mixture
Grade C evidence
Real Cochrane-reviewed trials in stroke and dementia; the current verdict is still "probably little or no benefit."
CTuftsin analog
Grade C evidence
Two real Russian comparator trials against benzodiazepines, never independently replicated in the West.
CGlutamate-cysteine-glycine
Grade C evidence
A real antioxidant tripeptide with thin efficacy evidence for the injectable route and a real, recent contamination track record.
CAcetyl octapeptide-3
Grade C evidence
Argireline's bigger sibling. The "63%" figure traces to the manufacturer's own testing, not an independent trial.
DEngineered IGF-1 analog
Grade D evidence
Zero published human clinical trials, per a dedicated 2026 review.
DPEGylated mechano growth factor
Grade D evidence
Real muscle-repair biology in animals; no human trial evidence that it works as sold.
AImcivree · MC4R agonist
Grade A evidence
The melanocortin-family drug with a real, current, genetic-testing-gated FDA approval for obesity — not a tanning or libido product.
ASandostatin · somatostatin analog
Grade A evidence
A real, decades-approved drug that suppresses GH — the mirror image of this site's GH-secretagogue cluster.
BSymlin · amylin analog
Grade B evidence
Cagrilintide's approved ancestor — real trial evidence, two decades on market, discontinued in 2025.
BByetta / Bydureon · GLP-1 agonist
Grade B evidence
The original approved GLP-1 drug, a decade before semaglutide — discontinued by its maker in October 2024.
CActhar Gel · ACTH
Grade C evidence
A real drug since 1952 — thin modern trial evidence for its priciest uses, and a real $260M+ federal enforcement record.
ALupron · GnRH agonist
Grade A evidence
Same GnRH mechanism and era as gonadorelin — but still in wide, current mainstream use rather than discontinued.
ADDAVP · vasopressin analog
Grade A evidence
A genuinely mainstream, decades-approved peptide drug — and a real hyponatremia risk worth knowing across every use.
DMitochondrial-derived peptide
Grade D evidence
Real biology, zero completed human trials — MOTS-c's sibling peptide, with the same evidence gap.
BMitochondria-targeted peptide
Grade B evidence
A real, narrow FDA approval in 2025 for Barth syndrome, alongside a real completed Phase 3 failure elsewhere.
AVoxzogo · CNP analog
Grade A evidence
A real, current, different-family FDA approval for achondroplasia — with a genuine, unresolved community debate about the goal itself.
AForteo · PTH(1-34)
Grade A evidence
Proved bone-building therapy works. Its boxed warning was removed by FDA in 2020 after 18 years of human surveillance found no cancer signal.
ATymlos · PTHrP analog
Grade A evidence
Teriparatide's approved cousin — an 86% vertebral-fracture reduction in its own placebo-controlled trial.
BMiacalcin / Fortical
Grade B evidence
A real, older bone drug whose role shrank after a 2013 FDA review found a real cancer signal in pooled trial data.
BFuzeon · HIV fusion inhibitor
Grade B evidence
The first approved peptide drug to block HIV by fusion inhibition — real efficacy, but a tolerability burden that pushed it to last resort.
BFirazyr · bradykinin B2 antagonist
Grade B evidence
A real rescue drug for hereditary angioedema attacks — though its very first pivotal trial missed against placebo.
BGlucaGen / Baqsimi / Gvoke
Grade B evidence
The real hormone that raises blood sugar. Its newest, easiest-to-use formulations rest on a thinner public trial record than most drugs here.
CNatrecor · BNP analog
Grade C evidence
A real drug whose risk-benefit picture genuinely shifted after approval — a large 2011 trial found no confirmed harm, but no real benefit either.
DHGF/c-Met pathway compound
Grade D evidence
Never tested in humans by the company that held its rights. Its closest tested relative failed two separate trials.
BBI 456906 · glucagon/GLP-1 agonist
Grade B evidence
A real, investigational dual agonist with published Phase 2 data and positive 2026 Phase 3 topline results, not yet approved anywhere.
AGattex/Revestive · GLP-2 analog
Grade A evidence
A real, approved GLP-2 analog for short bowel syndrome — the therapeutic opposite of the GLP-1 drugs covered elsewhere here.
ASomatuline Depot · somatostatin analog
Grade A evidence
Octreotide's approved sibling — same receptor class, monthly depot dosing, and a strong 2014 NEJM trial behind it.
BSignifor · somatostatin analog
Grade B evidence
Approved for Cushing's disease and acromegaly separately — but its own pivotal trial normalized cortisol in barely a quarter of patients.
AFirmagon · GnRH antagonist
Grade A evidence
The antagonist mirror image of leuprolide — no testosterone flare, non-inferior in its own pivotal trial.
ATrelstar/Triptodur · GnRH agonist
Grade A evidence
Leuprolide's close cousin, approved for both prostate cancer and, separately, precocious puberty.
AZoladex · GnRH agonist
Grade A evidence
Delivered as an implant, not an injection — and approved for prostate cancer, breast cancer, and endometriosis.
ACetrotide/Antagon · GnRH antagonists
Grade A evidence
The IVF antagonist pair — a real 2025 study found cetrorelix edges out ganirelix on OHSS risk despite similar live-birth rates.
BThe exact native peptide itself, approved for septic shock on a blood-pressure endpoint — not a proven mortality benefit.
BA synthetic amino-acid mixture for MS whose own FDA label admits the mechanism still isn't fully understood.
ANplate · Fc-peptide fusion
Grade A evidence
A "peptibody" for chronic ITP carrying a boxed warning about leukemia progression — in a population it isn't even approved to treat.
DOmontys · PEGylated peptide
Grade D evidence
Approved in 2012, recalled in 2013 after fatal anaphylaxis reports, formally withdrawn by the FDA in 2019.
ATrulicity · GLP-1 agonist
Grade A evidence
A major, still-marketed GLP-1 drug for diabetes — plus a real, separate FDA approval for cutting cardiovascular events.
BTanzeum · GLP-1 agonist
Grade B evidence
Discontinued in 2017 for business reasons — then vindicated by a positive cardiovascular trial published over a year later.
BAdlyxin · GLP-1 agonist
Grade B evidence
A genuinely neutral cardiovascular trial result, discontinued in the US in 2023 for commercial reasons.
ASomavert · GH receptor antagonist
Grade A evidence
Blocks growth hormone at the receptor rather than suppressing its release — a genuinely different acromegaly mechanism.
BMyalept · leptin analog
Grade B evidence
Real leptin-replacement evidence for an ultra-rare disease, approved without a placebo-controlled trial for ethical reasons.
ACortrosyn · ACTH(1-24)
Grade A evidence
A real diagnostic-only ACTH fragment used to test adrenal function — not a treatment for anything.
AChiRhoStim · diagnostic hormone
Grade A evidence
A real diagnostic peptide for pancreatic/gastrinoma testing — and a cautionary tale after years of unproven use as an autism treatment.
AKinevac · CCK fragment
Grade A evidence
A real diagnostic CCK fragment used to provoke gallbladder contraction during HIDA scans.
BTerlivaz · vasopressin analog
Grade B evidence
A genuinely recent (2022) approval for hepatorenal syndrome — real kidney benefit, boxed warning for respiratory failure.
ALong-acting oxytocin analog
Grade A evidence
Approved worldwide to prevent postpartum hemorrhage — genuinely still not FDA-approved in the US.
AKalbitor · kallikrein inhibitor
Grade A evidence
Treats the same condition as icatibant from the opposite direction — but its own anaphylaxis risk means a clinician must give it.
AAngiomax · leech-derived anticoagulant
Grade A evidence
A hospital-only anticoagulant engineered from leech-saliva peptide hirudin, used during coronary angioplasty.
AIntegrilin · snake-venom-derived peptide
Grade A evidence
A cyclic peptide antiplatelet drug derived from rattlesnake venom — its brand is gone, but the generic remains widely available.
ACubicin · lipopeptide antibiotic
Grade A evidence
A membrane-disrupting antibiotic inactivated by lung surfactant — it cannot treat pneumonia no matter how susceptible the bacteria are.
AGlycopeptide antibiotic
Grade A evidence
Approved in 1958, before modern efficacy trials existed — still the frontline MRSA drug despite real emerging resistance.
BCyclic peptide antibiotic
Grade B evidence
Discovered from an infected childhood wound in 1945 — now topical-only after systemic use proved significantly toxic to the kidneys.
BPolymyxin E · lipopeptide antibiotic
Grade B evidence
A revived last-resort antibiotic — and the 2015-2016 discovery of transferable resistance (mcr-1) was a real global-health alarm.
BLipopeptide antibiotic
Grade B evidence
Colistin's close cousin, given as the active drug directly rather than a prodrug — also the polymyxin in Neosporin and Polytrim.
ALinzess · GC-C receptor agonist
Grade A evidence
A bacterial-toxin-derived peptide for IBS-C/constipation, carrying a precise pediatric boxed warning traced to a specific preclinical finding.
ATrulance · GC-C receptor agonist
Grade A evidence
Linaclotide's cousin, modeled on the body's own uroguanylin rather than a bacterial toxin — same indications, same pediatric warning.
BIncrelex · recombinant IGF-1
Grade B evidence
Bypasses the GH axis entirely for a rare pediatric growth-failure disease — approved without a randomized control group.
ASupprelin LA/Vantas · GnRH agonist implant
Grade A evidence
The same GnRH-agonist implant sold under two brands for two different patients — one of which was discontinued in 2021.
ANatpara (discontinued) · Yorvipath
Grade A evidence
The first hypoparathyroidism drug, withdrawn over a cartridge defect — succeeded by a re-engineered PEGylated replacement.
BVasostrict · native ADH
Grade B evidence
The real, unmodified hormone itself, used for vasodilatory shock — its own major trial produced a genuinely neutral result.
DUrodilatin analog
Grade D evidence
A large, clean, well-designed heart-failure trial that missed both co-primary endpoints — never approved anywhere.
CRecombinant ANP
Grade C evidence
Approved in Japan since 1995 without a modern-style outcomes trial — real-world data has since raised a mortality concern.
AActhrel · ovine CRH analog
Grade A evidence
A real diagnostic hormone for Cushing's syndrome workups, discontinued in 2020 — its absence measurably hurt diagnostic accuracy.
AGonal-F · Follistim/Puregon
Grade A evidence
Recombinant FSH for IVF and ovulation induction, approved 1997 — manufactured rather than urine-purified, same biological effect.
BMenopur · historically Pergonal
Grade B evidence
Urinary-derived FSH/LH-like fertility drug with a remarkable origin story — purified from postmenopausal urine collected across Italian convents.
BRecombinant LH for profound LH-deficient IVF patients — approved 2004, formally withdrawn 2016 after the required confirmatory trial never happened.
ARecombinant hCG trigger shot for IVF, approved 2000 — mechanistically identical to urinary hCG, manufacturing consistency is the real difference.
ARecombinant TSH for thyroid cancer follow-up, letting patients stay on thyroid medication instead of becoming deliberately hypothyroid for testing.
BSemi-synthetic vancomycin descendant with a dual mechanism and a three-part boxed warning, including teratogenicity shown in animal studies.
ALong-half-life lipoglycopeptide antibiotic — an entire treatment course delivered in just one or two IV doses instead of days of infusions.
ASingle-dose lipoglycopeptide antibiotic with activity against vancomycin-resistant enterococci and a real lab-interference quirk on clotting tests.
BEpogen · Procrit
Grade B evidence
Recombinant erythropoietin approved 1989 — a landmark early biotech drug, now carrying a significant 2007 boxed warning after normalization trials backfired.
BLonger-acting engineered erythropoietin analog — subject to the same 2007 boxed warning, with its own trial (TREAT) finding a doubled stroke risk.
ARecombinant G-CSF that reduces infection risk during chemotherapy — its 2015 biosimilar, Zarxio, was the first biosimilar ever approved in the US.
APEGylated filgrastim engineered so its own clearance is regulated by neutrophil count — one dose per chemo cycle instead of daily injections.
APrialt · cone-snail venom peptide
Grade A evidence
A non-opioid painkiller derived from cone-snail venom, delivered only by implanted spinal pump for severe chronic pain.
AA peripherally restricted kappa-opioid peptide for hemodialysis itch, engineered to avoid crossing into the brain.
AAn oral ghrelin-receptor peptide used as a one-time diagnostic test for adult growth hormone deficiency, not a treatment.
BTractocile · oxytocin-receptor blocker
Grade B evidence
A mainstream labor-delaying drug across the EU and Canada since 2000 — never FDA-approved, after a US trial's fetal-death signal.
AThe first FDA-approved treatment for Rett syndrome, a synthetic analog of a natural IGF-1 breakdown fragment.
ANasal-spray GnRH agonist for endometriosis and central precocious puberty — same class as leuprolide, delivered without an injection.
ARecombinant hirudin analog for DVT prevention after hip replacement — real trials found it beat both heparin and enoxaparin, no longer marketed in the US.
BThe first approved HIT-specific anticoagulant, discontinued in 2012 for commercial reasons — not the safety failure some assume.
ARecombinant GM-CSF approved for bone marrow recovery in 1991, then for acute radiation syndrome in 2018 based entirely on animal studies.
BSuprefact/Suprecur · GnRH agonist
Grade B evidence
Same mechanism as leuprolide, used in one of the original 1982 trials for the drug class — never FDA-approved, apparently for market-timing reasons.
BGlycopeptide antibiotic
Grade B evidence
Vancomycin's Cochrane-reviewed cousin — equally effective and less nephrotoxic, reviewed by an FDA panel in the 1990s, still never approved in the US.
ACaspofungin / Micafungin / Anidulafungin
Grade A evidence
Three approved antifungals blocking a fungal-only cell-wall target — and a real case of the FDA and EMA reading the same rat-tumor data differently.
AParsabiv · calcimimetic
Grade A evidence
A peptide that lowers parathyroid hormone by the opposite mechanism from this site's PTH-analog drugs — three Phase 3 trials, over 1,700 patients.
ALutathera · radiolabeled somatostatin analog
Grade A evidence
Octreotide's radioactive cousin — a real cancer treatment, and the first peptide-receptor radionuclide therapy ever approved.
AZilbrysq · complement C5 inhibitor peptide
Grade A evidence
A once-daily self-injected peptide doing an antibody drug's job — real Phase 3 data, and the same boxed warning every C5 inhibitor carries.
AHumulin and analogs
Grade A evidence
The first genetically-engineered drug ever approved (1982) — and the peptide behind the biggest US drug-pricing story of the last decade.
BSurfaxin · KL4/sinapultide surfactant peptide
Grade B evidence
The first synthetic peptide-containing lung surfactant approved — then withdrawn commercially in 2015, for business reasons, not safety.
ANgenla · CTP-fusion growth hormone
Grade A evidence
The third once-weekly growth hormone on this site — approved in Canada well before the FDA, and still pediatric-only after its own adult trial missed its primary endpoint.
AVyvgart · FcRn-blocking antibody fragment
Grade A evidence
Clears out myasthenia gravis's disease-driving antibodies wholesale — approved 2021, then expanded into CIDP (2024) and every gMG antibody subtype (2026).
AAwiqli · once-weekly basal insulin
Grade A evidence
The first once-weekly basal insulin approved anywhere — cleared in Switzerland, Canada and the EU in 2024, then rejected and finally approved by the FDA almost two years later, for type 2 diabetes only.
AAphexda · CXCR4-antagonist cyclic peptide
Grade A evidence
Solves a real stem-cell-collection bottleneck for multiple myeloma transplant patients — 92.5% hit the collection target on it versus 26.2% on placebo. FDA-approved 2023.
ATresiba · ultra-long-acting multihexamer insulin
Grade A evidence
A real cardiovascular signal triggered a 2013 FDA rejection and a dedicated 7,637-patient outcomes trial — approved 2015 after the trial found no added risk and 40% less severe hypoglycemia.
BDual GLP-1/glucagon receptor agonist
Grade B evidence
A real approved medicine in China (2025) on strong Phase 3 data — but not approved anywhere in the West, and already named in this site's own FDA warning letters to unauthorized US sellers.
DGLP-1 agonist (discontinued)
Grade D evidence
Real Phase 3 efficacy data across 6,000+ patients — halted in 2010 by a hypersensitivity/anaphylaxis signal before ever reaching market.
CMK-677 · ghrelin-receptor agonist (non-peptide)
Grade C evidence
A 2011 hip-fracture trial was stopped early for heart failure — yet the same molecule is now in an actively-recruiting Phase 3 pediatric trial under a different name.
AEmpaveli/Syfovre · compstatin-derived C3 inhibitor
Grade A evidence
One active ingredient, three real FDA approvals in three unrelated diseases (2021, 2023, 2025) — plus a genuine post-marketing retinal-safety signal in the eye-injection formulation.
DRLX030 · recombinant relaxin-2 (discontinued)
Grade D evidence
A striking mortality signal in its first Phase 3 trial wasn't enough for FDA approval — the larger confirmatory trial regulators required came back negative, ending development.
ARuconest · recombinant C1 esterase inhibitor
Grade A evidence
An FDA-approved hereditary angioedema drug purified from transgenic rabbits' milk — approved for treating attacks, rejected in 2018 for preventing them.
APlasma-derived C1 esterase inhibitors
Grade A evidence
The two original human-plasma HAE replacement drugs, approved a year apart for opposite jobs — Cinryze (2008) to prevent attacks, Berinert (2009) to treat one already underway.
ABotox and four other approved brands
Grade A evidence
A real, 1989-approved protein drug with a dozen-plus indications — and two separate CDC-documented 2024-2025 outbreaks tied to counterfeit product and online self-injection, not the approved drug itself.
ACerezyme · recombinant glucocerebrosidase
Grade A evidence
A 1994-approved Gaucher disease enzyme drug whose only factory got shut down by a 2009 virus contamination — triggering a years-long shortage, a $175 million FDA penalty, and a permanent shift of patients to newer rivals.
AVPRIV & Elelyso · Gaucher enzyme replacements
Grade A evidence
Two competing Gaucher disease drugs fast-tracked to market by the Cerezyme shortage — one grown in human cells, the other the first plant-cell-expressed drug the FDA had ever approved.
AKrystexxa · PEGylated recombinant uricase
Grade A evidence
An enzyme humans lost through evolution, restored by infusion for severe gout — until antibodies against its own PEG coating stop it working, a problem a 2022-approved methotrexate combination directly addresses.
AFabrazyme · recombinant alpha-galactosidase A
Grade A evidence
A 2003-approved Fabry disease enzyme drug hit by the same 2009 Genzyme plant contamination as Cerezyme — but rationed to about 30% dose for nearly three years, and not converted from accelerated to full FDA approval until 2021.
AElfabrio · plant-cell-grown, PEGylated alpha-galactosidase A
Grade A evidence
A third Fabry disease enzyme, grown in plant cells and PEGylated for a longer half-life — a 2021 rejection over a COVID-blocked factory inspection delayed its 2023 approval by two years.
AMyozyme & Lumizyme · recombinant acid alpha-glucosidase
Grade A evidence
Turned uniformly fatal infantile Pompe disease into a treatable one — then got approved a second time under a different brand name because a bigger factory counted as a new product, with a restricted-use program not lifted until 2014.
ANexviazyme · glycoengineered acid alpha-glucosidase
Grade A evidence
A second-generation Pompe enzyme carrying substantially more of the uptake tag muscle cells use to absorb it — approved for adults in 2021, with an infant indication still pending FDA filing in 2026.
AAldurazyme · recombinant alpha-L-iduronidase
Grade A evidence
The first MPS I treatment, approved 2003 — a unanimous FDA advisory vote still wasn't enough to skip a further three-month delay, and it reached market through an unusual 50/50 joint venture between two separate biotechs.
AElaprase · recombinant iduronate-2-sulfatase
Grade A evidence
A real 2006 Hunter syndrome treatment that can't cross the blood-brain barrier — a decade-long, injected-into-the-spine version built to fix that failed its pivotal trial in 2017 and was discontinued in 2022.
APombiliti & Opfolda · engineered GAA plus oral stabilizer
Grade A evidence
A fourth Pompe disease enzyme whose own pivotal trial missed its primary endpoint for statistical significance — approved anyway on secondary evidence, then delayed a year by a COVID-blocked factory inspection, not the data.
AHumalog · reversed-sequence rapid-acting analog
Grade A evidence
The first insulin analog ever approved (1996) — and the single insulin product most tied to the US pricing crisis, from a $21 launch price to a 2017 rationing death that became a state law.
ANovoLog/Fiasp · aspartic-acid rapid-acting analog
Grade A evidence
A second, independently engineered rapid-acting analog approved in 2000 — now the first insulin with real 2025 biosimilar competition, including the first interchangeable rapid-acting insulin biosimilar.
ALantus/Toujeo · precipitating basal analog
Grade A evidence
The first long-acting basal insulin analog (2000) — its patent litigation against Eli Lilly and Merck shaped how insulin biosimilars reached the US market at all.
APulmozyme · recombinant human DNase I
Grade A evidence
A 1993-approved cystic fibrosis enzyme that succeeded where a 1958 bovine version failed on allergic grounds — and still has no FDA-approved biosimilar over a decade after its US patents expired.
AElitek · recombinant fungal urate oxidase
Grade A evidence
Clears a cancer-emergency uric acid surge within hours — but carries three boxed warnings, and real-world practice has shifted toward off-label doses far below the original FDA label.
AVimizim · recombinant N-acetylgalactosamine-6-sulfatase
Grade A evidence
The first MPS IVA treatment, approved 2014 — its own pivotal trial found the weekly dose worked and the every-other-week dose didn't, and a $380,000 list price took a five-year UK managed-access deal to resolve.
ANaglazyme · recombinant N-acetylgalactosamine 4-sulfatase
Grade A evidence
The first MPS VI treatment, approved 2005 — a real placebo-controlled trial win, later extended down to infants as young as three months in a dedicated follow-on study.
AMepsevii · recombinant beta-glucuronidase
Grade A evidence
The first MPS VII treatment, approved 2017, for a disease with perhaps 200 known patients worldwide — developed by Ultragenyx, not the BioMarin behind every other MPS drug on this site.
AWinrevair · ActRIIA-Fc activin trap
Grade A evidence
A first-in-class PAH drug approved 2024 — its pivotal trial cut death or clinical worsening by 84%, and a second 2025 trial expanded the label to the highest-risk patients.
AEnbrel · TNF receptor-Fc fusion
Grade A evidence
One of the first TNF-blocker biologics, approved 1998 — FDA-approved US biosimilars have existed since 2016 without ever reaching a patient, blocked by patents until 2029.
AEylea / Eylea HD / Zaltrap · VEGF-trap fusion
Grade A evidence
One fusion protein sold as three separate approved drugs since 2011 — now the subject of a live, multi-front biosimilar patent war with one at-risk launch already underway.
AActivase/Cathflo Activase · recombinant tPA
Grade A evidence
The original recombinant clot-buster, approved since 1987 — its own FDA stroke label still stops at 3 hours even though guidelines have endorsed 4.5 hours for over a decade.
BRetavase · engineered tPA deletion mutant
Grade B evidence
Alteplase re-engineered for two-bolus dosing — never beat alteplase's own survival numbers for heart attack, but a 2024 Chinese trial found it wins for a disease it isn't approved to treat: stroke.
ATNKase · engineered tPA, single-bolus
Grade A evidence
Approved for heart attack in 2000 on a 17,000-patient trial — then became the first new FDA-approved stroke medicine in nearly 30 years, in March 2025.
BStreptase · bacterial plasminogen activator
Grade B evidence
The original thrombolytic, approved 1977 — no longer sold in the US or Canada, displaced once an accelerated tPA regimen beat it on survival in 1993.
BAbbokinase/Kinlytic · human plasminogen activator
Grade B evidence
Approved 1978, pulled in 1999 over unscreened contaminated kidney cells, reapproved 2002 — off the US market again, with a funded 2028 comeback plan underway.
BEminase · chemically-blocked streptokinase complex
Grade B evidence
Approved 1989, roughly halved 30-day mortality against placebo — displaced within a decade once bolus-dosed tPA drugs matched its convenience without the antigenicity.
AMozobil · CXCR4-antagonist stem-cell mobilizer
Grade A evidence
The original CXCR4 antagonist, discovered as an anti-HIV drug before an unrelated side effect redirected it into cancer supportive care — approved 2008, now generic since 2023.
AEnjaymo · C1s-blocking monoclonal antibody
Grade A evidence
The first approved drug for cold agglutinin disease, a rare autoimmune anemia — a 2020 rejection was over a factory inspection, not the drug's own data, and a 2024 study found it isn't cost-effective at its current price.
AKanuma · recombinant lysosomal acid lipase
Grade A evidence
The first LAL deficiency treatment, approved 2015 for both the rapidly fatal infant form and the later-onset form — purified from the eggs of genetically engineered chickens, only the second FDA-approved drug ever made this way.
AXenpozyme · recombinant acid sphingomyelinase
Grade A evidence
The only approved ASMD (Niemann-Pick B) treatment — first cleared in Japan in 2022 under a pioneer fast-track designation, months before the FDA; the label explicitly excludes the disease's neurological form, which the enzyme can't reach.
APalynziq · PEGylated bacterial phenylalanine ammonia lyase
Grade A evidence
A PKU drug that substitutes an entirely different bacterial enzyme rather than restoring the missing human one — approved 2018 with a boxed warning and mandatory REMS for anaphylaxis, extended to adolescents only in February 2026.
ALevemir · myristic-acid-acylated basal insulin analog
Grade A evidence
The first engineered insulin to use reversible albumin binding, and the only basal insulin ever granted FDA pregnancy category B — discontinued in the US entirely through 2024 despite that record.
BExendin-4/Fc-fusion GLP-1 receptor agonist
Grade B evidence
Sanofi walked away from launching it in 2019, before its own pivotal trial reported a real 27% cardiovascular-risk reduction. Now being repositioned for a South Korean obesity filing, with no US or EU application pending.
AZegalogue · stabilized glucagon analog
Grade A evidence
A chemically restructured glucagon, not a reformulation of the native hormone — approved 2021, and now also being tested as the glucagon half of a dual-hormone artificial pancreas.
BFGF21 analog (Fc-fusion)
Grade B evidence
Missed its own primary endpoint in a Phase 2b cirrhosis trial, then reported a real 96-week cirrhosis-reversal signal — real enough that Novo Nordisk paid up to $5.2 billion for it in December 2025.
BFGF21 analog (glycoPEGylated)
Grade B evidence
Cleanly met both primary endpoints in a Phase 2b trial and holds FDA Breakthrough Therapy status since 2023 — now a Roche asset after a deal worth up to $3.5 billion closed October 2025.
BFGF21 analog (once-monthly, formerly BOS-580)
Grade B evidence
GSK's $2 billion once-monthly FGF21 bet just won FDA Breakthrough and EMA PRIME status in April 2026 — on the smallest completed trial of this site's three FGF21 analogs.
AFoundayo · non-peptide oral GLP-1 agonist
Grade A evidence
The first FDA-approved GLP-1 drug that's a small molecule, not a peptide — approved 1 April 2026, MHRA-approved in the UK by August 2026, still pending in the EU, Australia and Canada.
CPF-06882961 · non-peptide oral GLP-1 agonist
Grade C evidence
Pfizer's competing oral GLP-1 pill — real Phase 2 weight-loss data, discontinued in April 2025 after a liver-injury signal, following a related Pfizer candidate stopped the same way in 2023.
APluvicto · PSMA-targeted radioligand therapy
Grade A evidence
This site's second radioligand cancer therapy after Lutathera — approved 2022 on a real survival benefit, then expanded twice more (2025, 2026) after a 2022-2023 supply crisis got fixed.
ARyzneuta · non-PEGylated Fc-fusion G-CSF
Grade A evidence
A fourth engineered G-CSF joining this site's filgrastim/pegfilgrastim cluster — approved 2023 on a placebo-controlled trial and a head-to-head noninferiority win against pegfilgrastim.
AKineret & Arcalyst · IL-1 receptor antagonist and IL-1 trap
Grade A evidence
Two unrelated companies' answers to blocking interleukin-1 — approved seven years apart, then both approved for the identical ultra-rare disease one week apart in December 2020.
ATakhzyro & Andembry · plasma-kallikrein and Factor XIIa antibodies
Grade A evidence
Two more ways to block hereditary angioedema's bradykinin cascade — lanadelumab (2018) and garadacimab, approved in five other countries before the FDA cleared it in June 2025.
ARezzayo · once-weekly echinocandin antifungal
Grade A evidence
A fourth echinocandin engineered for once-weekly dosing — approved 2023 on a single pivotal trial and a narrower label than its class predecessors, after the FDA flagged a neurotoxicity signal.
AMimrylo · hepcidin-mimetic peptide
Grade A evidence
A genuinely new drug class — a synthetic hepcidin mimetic that restricts iron for erythropoiesis, approved 2026 for polycythemia vera on a real placebo-controlled Phase 3 trial.
BOnswik · once-weekly Fc-fusion basal insulin
Grade B evidence
Lilly's once-weekly basal insulin, built as an antibody Fc fusion instead of an albumin binder — FDA-approved 23 September 2026 for adults with type 2 diabetes only.
ASoliris & Ultomiris · complement C5 monoclonal antibodies
Grade A evidence
The original antibody drugs this site's own pegcetacoplan and zilucoplan pages reference — plus an SEC bribery settlement, a $125M investor settlement, and 2024's first biosimilars.
AOrladeyo · oral plasma kallikrein inhibitor
Grade A evidence
The one oral drug in this site's hereditary angioedema cluster — a once-daily pill approved 2020 on a real placebo-controlled trial, versus five injectable alternatives.
AAltuviiio & Hemlibra · factor VIII replacement and bispecific antibody
Grade A evidence
Two opposite fixes for hemophilia A's missing clotting factor — an engineered factor VIII built to break a real half-life ceiling (2023), and a bispecific antibody that skips factor VIII entirely, working even with inhibitors (2017).
AVabysmo · VEGF-A/Ang-2 bispecific antibody
Grade A evidence
The first bispecific antibody built for the eye, approved 2022 — matched this site's own aflibercept on vision outcomes with fewer injections, plus a real post-marketing eye-inflammation signal.
AApidra · zinc-free rapid-acting insulin analog
Grade A evidence
The third rapid-acting insulin analog, approved 2004 — the only one formulated without zinc, with no pricing scandal attached, just a real 70% US list-price cut in 2024.
APiasky · complement C5 monoclonal antibody
Grade A evidence
A third anti-C5 antibody for PNH, approved 2024 — engineered for once-monthly home self-injection instead of clinic infusion, matching eculizumab in two head-to-head Phase 3 trials.
AStrensiq · bone-targeted enzyme replacement
Grade A evidence
The first treatment for hypophosphatasia, approved 2015, with a real survival benefit against historical controls — and a roughly $285,000-a-year price that a UK regulator initially refused to cover.
AAlhemo & Hympavzi · anti-TFPI monoclonal antibodies
Grade A evidence
A genuinely new hemophilia drug class from two rival companies — both approved late 2024, both since expanded to cover more patients, and a real 2020 clinical hold that paused concizumab's trials after thrombotic events.
ALamzede · recombinant alpha-mannosidase enzyme replacement
Grade A evidence
The only approved treatment for alpha-mannosidosis — a clean 2018 biomarker win in the EU, a motor-function endpoint that fell short of significance, and a nearly five-year wait before the FDA approved it too.
AReplagal · recombinant alpha-galactosidase A enzyme replacement
Grade A evidence
A second Fabry disease enzyme, EU-approved in 2001 — but the one drug in this site's Fabry cluster never approved in the US, after Shire withdrew its FDA application in 2012 over a demand for more controlled trials.
AVeopoz · complement C5 monoclonal antibody
Grade A evidence
A fourth anti-C5 antibody, approved 2023 for CHAPLE disease — an ultra-rare complement disorder so uncommon the FDA accepted a 10-patient, placebo-free trial as its entire evidence base.
ARystiggo · FcRn-blocking monoclonal antibody
Grade A evidence
A second FcRn blocker for myasthenia gravis, approved 2023 — the first FDA-approved gMG drug covering both AChR- and MuSK-antibody-positive patients from day one.
CPlenaxis · GnRH antagonist
Grade C evidence
The first GnRH antagonist ever approved, 2003 — restricted to a mandatory allergic-reaction-monitoring program from day one, and discontinued from US sale by 2005.
AElonva · sustained recombinant FSH
Grade A evidence
One injection replaces the first seven days of daily FSH shots in IVF, EU-approved since 2010 — but never approved by the FDA after a 2014 rejection.
AEkterly · oral plasma kallikrein inhibitor
Grade A evidence
This site's HAE cluster's missing on-demand oral drug — approved July 2025 as the first pill for treating an attack already underway, not just preventing one.
AProlia & Xgeva · RANKL monoclonal antibody
Grade A evidence
This site's bone cluster's missing modern standard — a real 68% vertebral-fracture reduction in its 2009 pivotal trial, and a January 2024 boxed warning after dialysis-registry data found severe hypocalcemia in 41% of treated patients.
AEvenity · sclerostin monoclonal antibody
Grade A evidence
The one drug in this site's bone cluster that builds and preserves bone at once — approved in Japan first, then delayed over a year in the US after a real cardiovascular signal forced a boxed warning.
AImaavy · FcRn-blocking monoclonal antibody
Grade A evidence
A third FcRn blocker for myasthenia gravis, approved 2025 for the broadest antibody-positive population yet — then, in August 2026, the first drug ever approved for a second, unrelated disease: warm autoimmune hemolytic anemia.
BALT-801 · GLP-1/glucagon dual agonist
Grade B evidence
A real, investigational dual agonist in three separate programs — obesity, MASH, and alcohol use disorder — with published Phase 2 data in the first two and a positive July 2026 topline readout in the third.
BMariTide · GIPR-antagonist antibody/GLP-1 peptide conjugate
Grade B evidence
A real, investigational once-monthly obesity drug built on a genuinely new modality — up to 20% weight loss in its published Phase 2 trial, and a real tolerability problem that forced Amgen to redesign its Phase 3 dosing.
BNovo Nordisk · unimolecular GLP-1/amylin dual agonist
Grade B evidence
A real, investigational single-molecule GLP-1/amylin dual agonist in both injectable and oral form, with published Phase 1 data — but earlier in development than this site's other 2026 GLP-1-family additions.