→ Pro­tal­ix Bio­Ther­a­peu­tics has filed a BLA for their Fab­ry Dis­ease treat­ment, pe­gu­ni­gal­si­dase al­fa. The drug is a re­com­bi­nant en­zyme grown in plant cell cul­tures and de­signed to sup­ple­ment the lyso­so­mal en­zyme that is de­fi­cient in pa­tients with the ge­net­ic dis­or­der. Chiesi Glob­al Rare Dis­eases has signed up to com­mer­cial­ize the drug, in a deal that’s worth up to $760 mil­lion. A phase III study showed the Is­raeli biotech’s drug led to much high­er con­cen­tra­tion of en­zyme than Fab­razyme, the long­time stan­dard-of-care from Sanofi Gen­zyme.

→ Just days af­ter lin­ing up $145 mil­lion to pay for their first for­ay in­to the clin­ic, the Rally­bio team is go­ing back to Alex­ion for their lat­est hire. They’ve re­cruit­ed Róisín Arm­strong, who will head the de­vel­op­ment of RLYB211, a plas­ma-de­rived hy­per­im­mune glob­u­lin in de­vel­op­ment for the pre­ven­tion of fe­tal and neona­tal al­loim­mune throm­bo­cy­tope­nia. Arm­strong served as VP and Glob­al Med­i­cine Team leader at Alex­ion.

→.Macro­Gen­ics $MGNX was buoyed by the news that the FDA isn’t plan­ning on hold­ing an ex­pert com­mit­tee meet­ing to re­view its ap­pli­ca­tion for mar­ge­tux­imab. That re­moves one po­ten­tial bar­ri­er as they close in on a PDU­FA date of De­cem­ber 18.

→ Ver­sant Ven­tures, the Basel-based dis­cov­ery en­gine, an­nounced its lat­est com­pa­ny: Monte Rosa Ther­a­peu­tics. Backed with $32.5 mil­lion in Se­ries A funds, the com­pa­ny will work on de­vel­op­ing small mol­e­cules that de­grade dis­ease-caus­ing pro­teins, a rapid­ly grow­ing field.

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.”

The EU is reportedly working toward a centralized procurement deal for smallpox vaccines to tackle the rising number of cases of monkeypox, according to sources who spoke to the Financial Times.

Andrea Ammon, director of Europe’s CDC, told the FT that the European Commission is “definitely looking at what can be done centrally” and central procurement is “one of the options that are looked at but of course countries are also” looking into bilateral avenues.

More than 500 stakeholders sent comments to the FTC on whether the commission should look further into pharma middlemen, known as PBMs, with many of the commenters calling for more federal oversight.

Similar to the critical open comment period in a deadlocked FTC session last February, pharmacies and pharmacy groups are continuing to call out the lack of transparency among the top 3 PBMs, which control about 80% of the market.