European Medicine Agency (EMA)
The EMA’s Committee for Medicinal Products for Human Use (CHMP) held its monthly meeting from April 22nd to 25th. Here are the key outcomes:
Positive Opinions on New Medications:
- Altuvoct (efanesoctocog alfa, Sobi): Weekly treatment for hemophilia A. Sobi Press Release.
- Fruzaqla (fruquintinib, Takeda): For previously treated metastatic colorectal cancer. Takeda Press Release.
- Jeraygo (aprocitentan, Idorsia): For the treatment of resistant hypertension. Idorsia Press Release.
- Obgemsa (vibegron, Pierre Fabre Laboratories): For adults with overactive bladder syndrome.
- Truqap (capivasertib, AstraZeneca): For treating locally advanced or metastatic breast cancer with specific mutations.
The following biosimilars applications received positive opinions:
- Tofidence (tocilizumab, Biogen): A biosimilar to Genentech’s Actemra.
- Wezenla (ustekinumab, Amgen): A biosimilar to Johnson & Johnson’s Stelara.
CHMP recommended the approved of six label expansions:
- Alecensa (alectinib, Roche): For adjuvant treatment of resected non-small cell lung cancer.
- Opdivo (nivolumab, BMS): In combination with cisplatin and gemcitabine for first-line treatment of metastatic urothelial carcinoma.
- Rozlytrek (entrectinib, Roche) in patients older than 1 month with solid tumors that have an NTRK gene fusion.
- Rybrevant (amivantamab, Johnson & Johnson) as first-line combination therapy for non-small cell lung cancer (NSCLC).
- Sirturo (bedaquiline, Johnson & Johnson) in adults and children older than 5 years who weigh at least 15 kg with pulmonary tuberculosis due to Mycobacterium tuberculosis.
- Triumeq (dolutegravir / abacavir / lamivudine, ViiV Healthcare) for children 3 months and older weighing at least 6 kg with HIV-1.
For more detailed information about the CHMP meeting, see CHMP meeting highlights.
US Food and Drug Administration (FDA)
The FDA granted the following approvals:
1. Pivya (pivmecillinam, Utility Therapeutics) for the treatment of female adults with uncomplicated urinary tract infections (UTIs). FDA press release.
2. Anktiva (nogapendekin alfa inbakicept-pmln, Altor BioScience) for the treatment of BCG-unresponsive non-muscle invasive bladder cancer. FDA press release.
3. Lutathera (lutetium (177Lu) oxodotreotide, Novartis) for pediatric patients with gastroenteropancreatic neuroendocrine tumors. Novartis press release.
4. Ojemda (tovorafenib, Day One Biopharmaceuticals) accelerated approval for patients with relapsed or refractory BRAF-altered pediatric low-grade glioma. Day One press release.
5. Beqvez (fidanacogene elaparvovec-dzkt, Pfizer) for the treatment of adults with moderate to severe hemophilia B. Pfizer press release.
The FDA issued a complete response letter (CRL) to Abeona Therapeutics for its cell therapy, rademagene zamikeracel (pz-cel), for treating recessive dystrophic epidermolysis bullosa (RDEP). The CRL specified chemistry, manufacturing, and controls (CMC) issues that must be addressed. Abeona press release.
New data from clinical trials (Phase 2, Phase 3)
Here are the recent updates on Phase 2 and Phase 3 clinical trials with published data between the 22nd and 28th of April 2024:
1. Novartis: positive data from their phase 2/3 study of a new formulation for Coartem (artemether-lumefantrine) for the treatment of babies under 5 kg with malaria. Novartis press release.
2. Neurocrine Bioscience: positive results from their Phase 2 study of NBI-1065845 in adult patients with major depressive disorder (MDD). The study met its main goal as well as key secondary endpoints. Neurocrine press release.
3. Sanofi: rilzabrutinib has met its primary endpoint in the Phase 3 trial in heavily pre-treated immune thrombocytopenia (ITP) patients. Sanofi plans to file for approval in the US and the European Union in the second half of this year. Sanofi press release.
4. Aslan Pharmaceuticals: positive interim data from the Phase 2 study of eblasakimab for the treatment of adults with moderate-to-severe atopic dermatitis (AD). Aslan press release.
5. Lykos Therapeutics: completed a European Phase 2 study of midomafetamine (MDMA) capsules used in combination with psychological intervention for adults with post-traumatic stress disorder (PTSD), no specific data of the study are discolosed. Lykos press release.
6. Caliway Biopharmaceuticals: primary and secondary endpoints met in the Phase 2 trial of CBL-514 injection for cellulite treatment. Caliway press release.
Due to funding issues, Hepion Pharmaceuticals announced the decision to commence wind-down activities on the Phase IIb trial investigating rencofilstat for the treatment of non-alcoholic steatohepatitis (NASH). Hepion press release.
