The FDA has re­leased a clin­i­cal hold on Pfiz­er and Sang­amo’s he­mo­phil­ia A gene ther­a­py af­ter or­der­ing the com­pa­nies to stop the tri­al last year due to con­cerns about blood clots.

Just a week af­ter Pfiz­er an­nounced the FDA had un­leashed its gene ther­a­py for Duchenne mus­cu­lar dy­s­tro­phy from a clin­i­cal hold — which was paused as one pa­tient died and oth­ers ex­pe­ri­enced se­vere side ef­fects  — an­oth­er one of its gene ther­a­py pro­grams has been giv­en the green light to re­sume, Pfiz­er an­nounced dur­ing its Q1 earn­ings call yes­ter­day.

How­ev­er, the com­pa­ny an­tic­i­pates it won’t restart the Phase III tri­al un­til the third quar­ter of this year, Pfiz­er CSO Mikael Dol­sten not­ed dur­ing the meet­ing. In the mean­time, Pfiz­er needs to get ap­proval of their up­dat­ed study pro­to­cols from the FDA, ac­cord­ing to their Q1 earn­ings news re­lease.

Pfiz­er vol­un­tar­i­ly paused the study late last year af­ter pa­tients ex­pe­ri­enced ab­nor­mal­ly high lev­els of fac­tor VI­II, an es­sen­tial blood clot­ting fac­tor. In he­mo­phil­ia A, pa­tients lack a func­tion­al gene for mak­ing fac­tor VI­II, mean­ing that they have very low fac­tor VI­II lev­els at un­der 50%. In peo­ple with­out he­mo­phil­ia A, fac­tor VI­II lev­els usu­al­ly range be­tween 50% and 100%, but in the Pfiz­er tri­al, pa­tients had fac­tor VI­II of above 150%, putting them at risk for blood clots.

Soon af­ter Pfiz­er paused the tri­al, the FDA slapped on a clin­i­cal hold.

One pa­tient in the tri­al ex­pe­ri­enced deep vein throm­bo­sis be­low the knee, Pfiz­er not­ed in the news re­lease. That pa­tient had a his­to­ry of blood clots be­fore the tri­al be­gan, which is an ex­clu­sion cri­te­ria for par­tic­i­pat­ing in the study. How­ev­er, the pa­tient is now do­ing well, Pfiz­er said.

Now with the clin­i­cal hold gone, Pfiz­er and Sang­amo may have a chance to catch up to Bio­Marin, as the biotech plots to re­turn to the FDA with its gene ther­a­py this year. Bio­Marin’s gene ther­a­py was re­ject­ed by the FDA two years ago af­ter mul­ti­ple re­ports of fac­tor VI­II lev­els dwin­dling in pa­tients just years af­ter they re­ceived what is sup­posed to be a long-term fix.

Pfiz­er ex­pects a read­out from their he­mo­phil­ia A pro­gram some­time in the sec­ond half of 2023.

HHS on Thursday finalized its decision to withdraw a rule, proposed just before former President Donald Trump left office, that would’ve caused thousands of HHS and FDA regulations to automatically expire if they weren’t reviewed within two years, and every 10 years thereafter.

The decision follows the filing of a lawsuit last March, in which several nonprofits alleged that the outgoing administration planted “a ticking timebomb” for HHS, essentially forcing it to devote an enormous amount of resources to the unprecedented and infeasible task of reviewing thousands of regulations regularly.

At the end of January, the European Medicines Agency officially launched its new clinical trials info system (CTIS), although the migration to the new platform has only really just begun, and sponsors have until the end of January 2023 before all initial trial applications must be submitted through CTIS.

Overall, 56 clinical trial applications have been submitted in CTIS during the first 3 months since the launch of the system on Jan. 31, according to new data posted by the EMA. By comparison, about 4,000 new trials are authorized each year across Europe.

As concerns related to uptake and distribution continue to linger, Switzerland is among the first countries that plans to destroy hundreds of thousands of expired and unused Covid-19 vaccine doses.

The European country said it plans to destroy more than 600,000 doses of Moderna’s Spikevax Covid-19 vaccine as the doses have reached their expiration date.

However, Moderna CEO Stéphane Bancel told the World Economic Forum in Davos, Switzerland that he’s in the process of throwing 30 million doses in the garbage, exclaiming, “We have a big demand problem.”

As Neulasta sales slip, Amgen has yet another biosimilar to look out for: Amneal Pharmaceuticals and Kashiv Biosciences’ Flynetra.

Flynetra became the fifth approved biosimilar to Neulasta on Friday, snagging a win in neutropenia, a condition common among chemotherapy patients where neutrophils, a type of white blood cell that fights infection, are too low.

As of last summer, the list price of Neulasta was more than $6,400 per dose. It’s designed to be taken in a single dose per chemotherapy cycle. Amneal declined to reveal how much it intends to charge for Flynetra in an email to Endpoints News.