US FDA approval tracker: June
Last month the US regulator gave a controversial thumbs-up to Biogen’s Alzheimer’s drug Aduhelm, and the decision boosted the company’s market cap by $16.5bn. The approval was given on the condition that a confirmatory trial be run, but remarkably the study is not required to read out until 2030 at the latest. Aduhelm’s approval opened the floodgates in Alzheimer’s and Eli Lilly has confirmed it intends to file its similarly acting donanemab later this year. Elsewhere, three complete response letters were dished out, including one for Orphazyme’s arimoclomol in Niemann-Pick disease, with the FDA requesting further clinical data. The Pdufa for Ascendis’s paediatric growth hormone deficiency treatment TransCon hGH was extended; three other decisions were also delayed. The agency also missed the deadline for Abbvie's Rinvoq in two indications owing to ongoing safety concerns with the Jak class. Delays for other Jak inhibitors will likely continue into the next few months as the regulator continues to review the safety profile of Pfizer's Xeljanz (Go or no go? Ardelyx and Chemocentryx among those waiting FDA decisions, July 1, 2021).
| Notable first-time US approval decisions in June | ||||
|---|---|---|---|---|
| Project | Company | Indication(s) | 2026e sales by indication ($m) | Outcome |
| Aduhelm (aducanumab) |
Biogen/Eisai | Alzheimer's disease | 3,907 | Approved (accelerated) |
| Transcon hGH (lonapegsomatropin) |
Ascendis | Paediatric growth hormone deficiency | 1,407 | Extended to Sept 25 |
| PF-06482077/ 20vPnC/ Prevnar 20 |
Pfizer | Pneumonia | 1,037 | Approved |
| Lybalvi (ALKS 3831) |
Alkermes | Schizophrenia and bipolar I disorder | 380 | Approved |
| Eohilia/ TAK-721 |
Takeda | Eosinophilic esophagitis | 350 | No decision yet |
|
Brexafemme (ibrexafungerp/SCY-078) |
Scynexis | Vulvovaginal candidiasis | 302 | Approved |
| Miplyffa (arimoclomol) | Orphazyme | Niemann Pick disease type C | 297 | CRL (more data needed) |
| IV Tramadol | Avenue | Acute pain | 273 | CRL (second) |
| VP-102 | Verrica | Molluscum contagiosum | 271 | Extended to Sept 23 |
| Nexobrid | Mediwound/ Vericel |
Treatment of severe thermal burns | 165 | CRL (CMC) |
| Tanezumab | Pfizer/Lilly | Moderate-to-severe osteoarthritis pain | 155 | No decision yet |
| Ryplazim | Liminal | Congenital plasminogen deficiency | 66 | Approved |
| StrataGraft | Mallinckrodt | Adults with deep partial-thickness thermal burns | 59 | Approved |
| Rylaze (JZP-458) |
Jazz | Component of chemo regimen to treat ALL or lymphoblastic lymphoma in patients who are allergic to E. coli-derived asparaginase | 33 | Approved |
| Rezipres (Ephedrine injection/ET-203) | Eton/Sintetica | Hypotension | 37 | Approved |
| Verkazia | Santen | Keratoconjunctivitis in patients aged 4-18 | - | Approved |
| Tembexa (brincidofovir) |
Chimerix | Smallpox (adult and paediatric patients) | - | Approved (~1 month early) |
| Source: Evaluate Pharma & company releases. | ||||
| Advisory committee meetings in June | ||||||
|---|---|---|---|---|---|---|
| Project | Company | Adcom date | Indication | 2026e sales by indication ($m) | Outcome | Note |
| Retifanlimab (INCMGA0012) |
Incyte/Macrogenics | June 24 | Adult patients with locally advanced or met squamous carcinoma of the anal canal who have progressed on or who are intolerant of platinum-based chemotherapy. | 53 | 13-4 voted for confirmatory study | Anti-PD-1 MAb, Pdufa July 25 |
| Source: Evaluate Pharma & FDA adcom calendar. | ||||||
| Supplementary and other notable approval decisions in June | |||
|---|---|---|---|
| Product | Company | Indication (clinical trial) | Outcome |
| Rinvoq | Abbvie | Psoriatic arthritis (Select-PsA 1, Select-PsA 2) | Missed (FDA citing review of Pfizer's Xeljanz) |
| Rinvoq | Abbvie | Ankylosing spondylitis (Select-Axis 1) | Missed (FDA citing review of Pfizer's Xeljanz) |
| Ruxolitinib cream | Incyte | Atopic dermatitis (TruE-AD1, TruE-AD2) | Extended to Sept 21 |
| Jakafi | Incyte/Novartis | Paediatric patients with steroid-refractory GVHD (Reach3) | Extended to Sept 22 |
| Cosentyx | Novartis | Moderate-to-severe plaque psoriasis in children and adolescents aged 6 to <18 years (NCT03668613, NCT02471144) | Approved |
| Ultomiris | Astrazeneca | Children and adolescents with PNH | Approved |
| Trikafta | Vertex | Children aged 6 to 11 who have at least one F508del mutation in the CFTR gene or a mutation in the CFTR gene that is responsive based on in vitro data (NCT03691779) | Approved |
| Ayvakit | Blueprint | Advanced systemic mastocytosis (Pathfinder, Explorer) | Approved |
| Solosec | Lupin | Treatment of trichomoniasis in adults and adolescents | Approved |
| Wegovy (semgalutide 2.4mg) | Novo Nordisk | Obesity (Step 1, 2, 3, 4) | Approved |
| Epclusa | Gilead | Expansion of the paediatric indication for chronic hepatitis C (NCT03022981) | Approved |
| Pradaxa (oral pellets) | Boehringer Ingelheim | Blood thinner for children aged 3 months to less than 12 years old (NCT01895777, NCT02197416) | Approved |
| Alecensa | Roche | 1st-line Alk-positive NSCLC (Bfast) | No decision yet |
| Source: Evaluate Pharma & company releases. | |||
| FDA Covid-19 EUAs | |||
|---|---|---|---|
| Product | Company | 2026e sales by indication ($m) | Note |
| Actemra | Roche | - | Treatment of hospitalized adults and paediatric patients |
| Source: Evaluate Pharma & company releases. | |||
