US FDA approval tracker: August

Snippets

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.

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