Last year, the European Commission approved a record 55 therapies containing a new active substance, including 21 medicines for rare diseases. Among them were a number of gene therapies for indications such as cancer and hemophilia.
The number of medicines and vaccines containing a new active substance (NAS) approved in the EU hit a new high last year, with 55 products receiving a centralized marketing authorization, compared with 52 in 2021, which itself was a record number.
Orphan drug approvals also set a new benchmark in 2022, with 21 medicines for rare diseases accounting for more than a third of all NAS-containing products authorized for marketing by...
If US tariffs on pharmaceutical supply chain products come into force they could be disruptive for companies. Ewan Townsend, of the international law firm Arnold & Porter, explains how companies can mitigate issues through negotiating with suppliers and reallocating tariff responsibilities.
The European Medicines Agency says that a regulatory reliance pilot that it is supporting to speed up assessments of post-approval changes appears to be accelerating evaluation timelines in non-EU countries.
The UK regulator’s draft guideline on the use of external control arms based on real-world data reflects concepts similar to those outlined by the US Food and Drug Administration.
The Pink Sheet's list of EU centralized approvals of new active substances has been updated to include two new products, one of which is Vyjuvek, Krystal Biotech's advanced therapy for treating dystrophic epidermolysis bullosa.
Bridget Dooling, a law school professor who reviewed draft regulations from the FDA and other agencies as an OMB attorney, said prior federal court decisions suggest judges typically defer to agency decisions based in science.
The new CBER director, once best known in the pharma world for criticizing accelerated approval, committed to expediting cancer drugs with surrogate endpoints.
The agency said it will consider knee extensor muscle strength as a potential intermediate clinical endpoint to support accelerated approval, but Stealth CEO Reenie McCarthy said announcing it via complete response letter is confusing and inefficient.
