2024 FDA Approvals: Breakthroughs in Cancer and Rare Diseases
Cancer Treatments:
Durvalumab (Imfinzi, AstraZeneca):
Approved for adults with limited-stage small cell lung cancer (LS-SCLC) whose disease has not progressed following concurrent platinum-based chemotherapy and radiation therapy1.
Ensartinib (Ensacove, Xcovery Holdings, Inc.):
Approved for adult patients with anaplastic lymphoma kinase (ALK)-positive locally advanced or metastatic non-small cell lung cancer (NSCLC) who have not previously received an ALK-inhibitor1.
Zenocutuzumab-zbco (Bizengri, Merus N.V.):
Granted accelerated approval for adults with specific types of non-small cell lung cancer and pancreatic adenocarcinoma1.
Rare Diseases:
Zanidatamab-hrii (Ziihera, Jazz Pharmaceuticals, Inc.):
Approved for previously treated, unresectable or metastatic HER2-positive biliary tract cancer12.
Levacetylleucine (Aqneursa):
Approved for neurological manifestations of Niemann-Pick disease type C in adults and pediatric patients weighing at least 15 kg2.
Other Notable Approvals:
Rezdiffra (Madrigal Pharmaceuticals):
First-ever treatment approved for metabolic dysfunction-associated steatohepatitis (MASH)35.
Cobenfy (Bristol Myers Squibb):
First novel therapeutic approved for schizophrenia in 35 years3.
Vafseo (vadadustat):
Approved for the treatment of anemia due to chronic kidney disease in adults who have been receiving dialysis for at least 3 months2.
General Trends:
The FDA approved 50 novel drugs in 2024, with 24 being first-in-class2.
More than half of the oncology approvals were for biologics or biosimilars4.
Sources:
1. https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancerhematologic-malignancies-approval-notifications
2. https://www.fda.gov/media/184967/download
3. https://www.biospace.com/biospace-fda-decision-tracker
4. https://www.aptitudehealth.com/oncology-news/new-fda-and-ema-oncology-drug-approvals-in-q2-2024-largely-focused-on-biologics-and-specific-molecular-targets/