Shots:
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,…
Shots:
Royalty Pharma acquiring Revolution Medicines’ royalties for daraxonrasib (P-III RASolute 302), providing up to $1.25B across five $250M milestone tranches, plus an additional $750M debt facility in three tranches
Revolution received the $250M upfront, with future payments tied to P-III data (2026), FDA approval (by Jan 2028), and sales milestones, allowing access to further…
Shots :
Merck’s ENFLONSIA has been approved by the US FDA to prevent RSV lower respiratory tract disease in newborns and infants entering their first RSV season, offering protection for up to 5mos. with a single 105mg dose, regardless of weight
The approval was supported by the P-IIb/III (CLEVER) study of ENFLONSIA that demonstrated reduced…
Shots:
CoreMap received the US FDA’s IDE approval of CoreMap EP (Endocardial Electrophysiology) to extend the 3-phase INvENI clinical study into the US in pts with Atrial Fibrillation (AF) and long-standing persistent AF
The P-I study assessed acute safety of CoreMap EP in 14 pts while P-II study showed acute safety & effectiveness in 15 pts. The…
Shots:
PharmaShots’ Designation Report provides a concise overview of the latest drug designations granted by major regulatory authorities, including the FDA, EMA, MHLW, Health Canada and NMPA
The April 2025 report covers designations granted to 36 drugs and 6 medical devices, spanning 14 small molecules, 3 biologics, 11 cell and gene therapies & 6 medical devices among…
Shots:
InnoCare Pharma has approved Minjuvi (tafasitamab) + Lenalidomide, followed by Minjuvi monotx., for treating adult pts. with r/r diffuse large B-cell lymphoma (DLBCL) who are ineligible for autologous stem cell transplant (ASCT)
Tafasitamab, a humanized Fc-modified cytolytic CD19-targeting immunotherapy, combined with lenalidomide, is already approved for treating eligible DLBCL pts. in Hong Kong, Macau,…
Shots:
PharmaShots’ Designation Report provides a concise overview of the latest drug designations granted by major regulatory authorities, including the FDA, EMA, MHLW, and NMPA
The March 2025 report covers designations granted to 37 drugs and 3 medical devices, spanning 11 small molecules, 10 biologics, 10 cell and gene therapies & 3 medical devices among…
Shots:
The CHMP has issued a positive opinion recommending marketing authorization for Sephience (sepiapterin) to treat PKU in patients of all ages and disease severities, expected the EC approval within two mos., with the decision applying across the EU including Iceland, Norway, and Liechtenstein
Sepiapterin's NDA is under FDA review (PDUFA: July 29, 2025), and…
Shots:
TRIUMpH program consists of 2 P-III trials assessing tegoprazan in US GERD pts with erosive esophagitis (EE) & non-erosive reflux disease (NERD); EE’s maintenance phase to end by Q3’25, with FDA’s NDA filing for both EE & NERD planned in Q4’25
EE study (vs lansoprazole) in 1250 pts (incl. 463 with LA Grade C/D)…
Shots:
PharmaShots’ Designation Report provides a concise overview of the latest drug designations granted by major regulatory authorities, including the FDA, EMA, MHLW, and NMPA
The February 2025 report covers designations granted to 53 drugs and 4 medical devices, spanning 16 small molecules, 14 biologics, 18 cell and gene therapies & 4 medical devices among…

