US FDA Approval Tracker: August

September 03, 2020 US FDA approval tracker: August

Joanne Fagg

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

2026e sales by Project Company Outcome indication ($m)

Evrysdi (risdiplam) Roche/PTC 1,786 Approved

Jyseleca (filgotinib) Gilead/Galapagos 1,404 CRL

Roctavian (valoctocogene Biomarin 1,365 CRL roxaparvovec/valrox)

GSK/Seattle Blenrep (belantamab mafodotin) 1,262 Approved Genetics

Viltepso (viltolarsen) Nippon 585 Approved

Viaskin Peanut DBV 556 CRL

GSK/ Dostarlimab 516 No decision yet Anaptysbio

Winlevi Cassiopea 420 Approved (clascoterone cream 1%)

Enspryng (satralizumab) Roche 419 Approved

Sogroya Novo Nordisk 326 Approved (somapacitan/NN8640)

Positive adcom, Pdufa due Ryoncil (remestemcel-L) Mesoblast 274 Sep 30

Olinvyk (IV oliceridine) Trevena 226 Approved

Pedmark Fennec 226 CRL

Ryanodex Eagle 4 CRL

EM-100 (Ketotifen PF Ophthalmic Eton - Extended to Sep 15 Solution)

Veverimer Tricida - CRL

Xaracoll Innocoll - Approved

Delayed, no new action Tlando Lipocine - 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 Relapsing and remitting MS (Asclepios I (ofatumumab/Arzerra Novartis Approved and II) SC)

Bristol Paediatric patients with moderate to Stelara Approved Myers/J&J severe plaque psoriasis (Cadmus Jr)

HIV-1 infections in virologically Dovato GSK/Shionogi Approved suppressed adults (Tango)

Darzalex (+ Kyprolis Relapsed/refractory multiple myeloma J&J/Genmab Approved + dexamethasone) (Candor)

GSK/Innoviva/ Trelegy Ellipta Asthma (Captain) No decision yet Theravance

GSK/Innoviva/ Ad com voted 14-1 against Trelegy Ellipta COPD (Impact) Theravance 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.