US FDA approval tracker: August

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

Amarin forced to look to Europe as US patent hopes dashed

The 30% fall in Amarin’s shares yesterday, even before the company’s appeal hearing proper started, made it clear that few expected the group to hold off generic competition for Vascepa in the US. And now Amarin has officially lost its appeal, the group’s fate is largely down to European sales. One problem: Vascepa has yet to launch in Europe. The group only took the decision to go it alone on the continent in August and approval is not expected before early 2021. But launches in Europe are notoriously expensive, with slow ramp ups, due to the need to secure country-by-country pricing and distribution arrangements. It is also far from clear what the market opportunity is in Europe – much will depend on the price Vascepa can command. Leerink analysts had previously forecast $1bn in peak European sales, but looks ambitious, despite a heart benefit boost last year. Amarin also said it would be looking to approval in China, where it is partnered with Eddingpharm, and the Middle East to boost sales. However, despite the Amarin trying to put on a brave face shares fell another 4% this morning at the enormity of the news.

https://www.evaluate.com/vantage/articles/news/snippets/amarin-forced-look-europe-us-patent-hopes-dashed

Baxter nets FDA OK for new class of dialysis filter, closer to human kidney

Baxter has received a green light from the FDA for a new type of dialysis filter that sifts out a wider range of molecules from the blood compared to traditional membranes—including those associated with the heightened inflammatory and cardiovascular disease seen in patients suffering from kidney failure.

The Theranova cartridge is designed to be used with existing dialysis machines with little change in treatment, while offering a filtration profile that more closely mimics the natural kidney, the company said.

“Individually, the side effects from standard [hemodialysis], which patients typically undertake three days a week, four hours per day, may seem manageable,” said Mary Gellens, senior medical director at Baxter. “However, the chronic effects of treatment accumulate and over time, cause some patients to give up on therapy.”

The expanded hemodialysis therapy provided by Theranova clears out a group of larger, heavier chains of molecules that form uremic toxins, which are typically removed from the body through well-functioning kidneys. 

The dialyzer also allows the blood’s essential proteins to pass back into the patient and helps to maintain proper levels of albumin, which carry hormones and enzymes throughout the body.

Theranova was previously launched outside of the U.S. in 2016, and is currently available in 44 countries. The FDA granted Baxter a de novo clearance, a rarity for kidney failure treatments, establishing a new regulatory class of dialyzer.

https://www.fiercebiotech.com/medtech/baxter-nets-fda-de-novo-clearance-for-a-new-class-dialysis-filter-more-similar-to-human