Go or no go? Ardelyx and Chemocentryx among those waiting FDA decisions

A number of small biotechs are awaiting news on FDA approval in July, including Albireo and Chemocentryx, in a month that looks quiet on the regulatory front for larger developers. Fibrogen’s anaemia pill will go under the spotlight at a panel meeting in mid July, a project that has been dogged by safety concerns.

Big pharma has an interest in the ongoing regulatory delays for the Jak inhibitor class, however, where decisions on four projects are pegged for early in the third quarter. FDA opinions for Rinvoq, Olumiant, abrocitinib and Xeljanz are all due, from Abbvie, Eli Lilly and Pfizer, but with a safety review ongoing timings are hard to know.

Jak delay

Last week the Pdufas for Abbvie’s Jak inhibitor Rinvoq in psoriatic arthritis and ankylosing spondylitis were missed with no new date disclosed. Abbvie said that FDA is still reviewing Pfizer's post-marketing study, Oral Surveillance, that raised concerns about Xeljanz in rheumatoid arthritis and the class more broadly. 

Xeljanz already has a black-box warning for thrombosis and malignancies and data from Oral Surveillance, released in January, failed to show that the Pfizer drug was non-inferior to a TNF inhibitor on rates of major adverse cardiovascular events and cancers.

It is not known when the FDA's review will conclude. Abbvie has much riding on Rinvoq's label expansion, and plans to file in ulcerative colitis before the end of the year. Maintenance data released this week raised no new safety issues.  

Reputation and rare diseases

Roxadustat’s US advisory panel hearing is set for July 15, and a recent positive opinion in Europe could be considered encouraging. However, a previous admission of a major error in published data and the NDA filing means the FDA will be closely scrutinising the submission. 

Fibrogen needs roxadustat to be approved with a broad label without a black box warning of potential cardiotoxicity. That is far from guaranteed, however (Fibrogen stretches the bounds of credibility, April 7, 2021).

Albireo, meanwhile, could have the first bile acid transporter inhibitor on the market in the US; approval of odevixibat is being sought in progressive familial intrahepatic cholestasis (PFIC), a rare paediatric liver disorder.

PFIC is characterised by the build-up of bile in the liver and is usually treated surgically by partial external biliary diversion or liver transplant. There are a number of different types of disease, each characterised by specific gene mutations. Type 2 makes up around half of cases.

Albireo’s odevixibat aims to improve patients’ quality of life, in phase 3 odevixibat met both co-primary endpoints; the change in pruritus, and bile acid reduction in type 1 and 2 patients (Albireo sets the standard for Mirum to hit, September 8, 2020).

The ultimate goal is to prevent liver transplants, but it is too soon to know whether odevixibat can contribute to that outcome.

The table below lists first-time US approvals and advisory meetings due next month, with consensus forecasts from Evaluate Pharma.

This article has been updated to include anifrolumab's Pdufa.