Approvals

In 2023, the FDA greenlit 55 new drugs and 34 cell and gene therapies. Follow along as BioSpace keeps you up to date on all of the FDA’s decisions in 2024.
The FDA has followed in the footsteps of its European counterparts and granted accelerated approval to PTC Therapeutics’ gene therapy Kebilidi for AADC deficiency. It is the first approved gene therapy to be delivered directly to the brain.
Scemblix was granted accelerated approval by the FDA for the treatment of certain patients newly diagnosed with chronic myeloid leukemia. The expanded indication increases the eligible patient population by approximately four times, according to Novartis.
Orlynvah is the first oral penem approved in the U.S. and Iterum Therapeutics’ first FDA-approved product. CEO Corey Fishman said the company will renew its efforts to look for a potential partner to maximize value for its stakeholders.
RSV
Pfizer’s Abrysvo is the first respiratory syncytial virus vaccine that can be used for adults less than 50 years of age. Tuesday’s label expansion covers younger adults who are deemed at higher risk of RSV-related lower respiratory tract disease.
The regulator’s approval on Friday of Vyloy for gastric or gastroesophageal junction cancer makes it the first and only claudin 18.2–targeted therapy approved in the U.S. for these indications, according to Astellas.
The approval makes Pfizer’s Hympavzi the first once-weekly subcutaneous prophylactic injection for hemophilia B in the U.S., according to the company, which is currently embroiled in a row with activist investor Starboard Value.
With the regulatory approval for advanced breast cancer, Roche’s inavolisib is a potential challenger to Novartis’ PI3K inhibitor Piqray, which last year generated $505 million in revenue.
Opdivo’s approval for patients with resectable non-small cell lung cancer comes as the regulator recently raised concerns of overtreatment with this type of therapeutic regimen with platinum-doublet chemotherapy.
FDA
Already approved in six indications, Sanofi and Regeneron can now add chronic obstructive pulmonary disease to the list for their blockbuster injection.
PRESS RELEASES