The FDA bared its teeth last month, delivering knockbacks for Biomarin, Gilead and Galapagos that took investors by surprise. Biomarin’s market cap lost $7.5bn on the rejection of its haemophilia A gene therapy candidate Roctavian (valrox). There have been questions over the project’s durability, and the regulator demanded two years’ data from an ongoing phase III study. The FDA also requested additional data on Gilead and Galapagos’ filgotinib, a Jak1 inhibitor. Readout from two studies investigating the drug’s impact on testicular toxicity aren’t due until next year, pushing filgotinib even further behind in a crowded rheumatoid arthritis market. Elsewhere, a panel voted unanimously against adding a mortality benefit to the COPD label for GSK’s Trelegy Ellipta. Despite the setback analysts believe that exacerbation benefits already detailed on the label are sufficient to continue to drive uptake in COPD. The respiratory drug has other tricks up its sleeve – a regulatory decision in asthma is awaited and, while Trelegy’s sales are driven by COPD, an approval here would be an added bonus.
| Notable first-time US approval decisions in August | |||
|---|---|---|---|
| Project | Company | 2026e sales by indication ($m) | Outcome |
| Evrysdi (risdiplam) | Roche/PTC | 1,786 | Approved |
| Jyseleca (filgotinib) | Gilead/Galapagos | 1,404 | CRL |
| Roctavian (valoctocogene roxaparvovec/valrox) | Biomarin | 1,365 | CRL |
| Blenrep (belantamab mafodotin) | GSK/Seattle Genetics | 1,262 | Approved |
| Viltepso (viltolarsen) | Nippon | 585 | Approved |
| Viaskin Peanut | DBV | 556 | CRL |
| Dostarlimab | GSK/ Anaptysbio | 516 | No decision yet |
| Winlevi (clascoterone cream 1%) | Cassiopea | 420 | Approved |
| Enspryng (satralizumab) | Roche | 419 | Approved |
| Sogroya (somapacitan/NN8640) | Novo Nordisk | 326 | Approved |
| Ryoncil (remestemcel-L) | Mesoblast | 274 | Positive adcom, Pdufa due Sep 30 |
| Olinvyk (IV oliceridine) | Trevena | 226 | Approved |
| Pedmark | Fennec | 226 | CRL |
| Ryanodex | Eagle | 4 | CRL |
| EM-100 (Ketotifen PF Ophthalmic Solution) | Eton | – | Extended to Sep 15 |
| Veverimer | Tricida | – | CRL |
| Xaracoll | Innocoll | – | Approved |
| Tlando | Lipocine | – | Delayed, no new action date provided |
| Lampit (nifurtimox) | Bayer | – | Approved |
| Sources: EvaluatePharma, Go or no go? Valuable drugs set for FDA decisions, company releases. |
| Supplementary and other notable approval decisions in August | |||
|---|---|---|---|
| Product | Company | Indication (clinical trial) | Outcome |
| Kesimpta (ofatumumab/Arzerra SC) | Novartis | Relapsing and remitting MS (Asclepios I and II) | Approved |
| Stelara | Bristol Myers/J&J | Paediatric patients with moderate to severe plaque psoriasis (Cadmus Jr) | Approved |
| Dovato | GSK/Shionogi | HIV-1 infections in virologically suppressed adults (Tango) | Approved |
| Darzalex (+ Kyprolis + dexamethasone) | J&J/Genmab | Relapsed/refractory multiple myeloma (Candor) | Approved |
| Trelegy Ellipta | GSK/Innoviva/ Theravance | Asthma (Captain) | No decision yet |
| Trelegy Ellipta | GSK/Innoviva/ Theravance | COPD (Impact) | Ad com voted 14-1 against mortality benefit claim |
| Xolair | Novartis | Nasal polyps (Polyp 1 and 2) | No decision yet |
| Sources: EvaluatePharma, Go or no go? Valuable drugs set for FDA decisions, company releases. |
https://www.evaluate.com/vantage/articles/news/snippets/us-fda-approval-tracker-august
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