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The US FDA has approved label expansion of Alhemo (concizumab-mtci) to prevent or reduce the frequency of bleeding episodes in pts (≥12yrs.) with HA/HB without inhibitors
Approval was based on P-III (Explorer8) trial assessing Alhemo (n=42, 1mg/kg followed by 0.2mg/kg QD) vs no prophylaxis (n=21) in 156 males with HA/HB, where median duration of…
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Pfizer reported positive topline results from the P‑III (BASIS) study evaluating HYMPAVZI (marstacimab) in hemophilia A or B pts (N=48) with inhibitors, meeting the 1EP and demonstrating a 93% reduction in annualized bleeding rate (ABR) vs. on‑demand treatment (1.39 vs. 19.78; p < 0.0001) over 12mos.
HYMPAVZI, a once‑weekly SC therapy that targets TFPI,…
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Sangamo regains rights to giroctocogene fitelparvovec (gene therapy for hemophilia A) as Pfizer ends their collaboration (effective Apr 21, 2025) & decids not to file BLA or MAA to pursue commercialization. Sangamo plans to seek a new partner
The drug is in P-III (AFFINE) trial involving 75 adult males with severe hemophilia A…
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The EMA’s CHMP has granted positive opinions to 3 Biologics and 2 New Chemical Entities in November 2024, leading to treatments for patients and advances in the healthcare industry
The major highlighted drugs were AbbVie’s Elahere to treat Ovarian Cancer and Pfizer’s Hympavzi for Hemophilia A and B
PharmaShots has compiled a list of…
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The US FDA approved 5 NDAs and 2 BLA in July 2023, leading to treatments for patients and advances in the healthcare industry. The CDER and CBER approved 76 novel products in 2023
In July 2023, the major highlights drugs were Beyfortus (nirsevimab) approval for the prevention of RSV lower respiratory tract disease in…

