New Drug Designations – April 2025  

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…

ByRidhi Rastogi3 days ago

News

BioCryst Reports the US FDA’s NDA Acceptance and Priority Review of Orladeyo for Hereditary Angioedema (HAE)

Shots:The US FDA has accepted NDA & granted priority review to Orladeyo (berotralstat) for the treatment of HAE pts (2-11yrs.), with PDUFA action date of Sep 12, 2025. Line extension application has been filed with the EMA; further filings are planned globally incl. Japan & CanadaNDA was supported by P-III (APeX-P) trial assessing Orladeyo…

ByRidhi RastogiMay 16, 2025

News

Cytokinetics Reports P-III (MAPLE-HCM) Trial Data of Aficamten for Obstructive Hypertrophic Cardiomyopathy

Shots:Cytokinetics has reported P-III (MAPLE-HCM) trial data assessing aficamten vs SoC metoprolol in pts (n=175) with symptomatic obstructive hypertrophic cardiomyopathy (HCM); regulatory review is ongoing in the US FDA (PDUFA: Dec 26, 2025) & EMA, with its NDA also under NMPA's priority review The trial met its 1EP, showing superior improvement in peak oxygen…

ByRidhi RastogiMay 14, 2025

News

REGENXBIO Reports the US FDA’s BLA Acceptance and Priority Review of RGX-121 for Treating Mucopolysaccharidosis II

Shots:The US FDA has accepted BLA & granted priority review to RGX-121 (clemidsogene lanparvovec) for the treatment of Mucopolysaccharidosis II (MPS II or Hunter syndrome), with PDUFA action date of Nov 09, 2025As per Jan 2025 partnership, NS Pharma (Nippon Shinyaku’s subsidiary) will commercialize RGX-121 in the US post approval while REGENXBIO will handle…

ByRidhi RastogiMay 14, 2025

News

The US FDA Approves BMS’ Opdivo + Yervoy as 1L Treatment for Hepatocellular Carcinoma (HCC)

Shots:The US FDA has approved Opdivo + Yervoy as 1L therapy to treat inoperable or advanced HCC adultsApproval was based on P-III (CheckMate-9DW) trial assessing Opdivo (1mg/kg, Q3W) + Yervoy (3mg/kg, Q3W), followed by Opdivo alone (480mg, Q4W) vs lenvatinib/sorafenib in 668 pts with advanced HCC who had not received prior systemic therapy.…

ByRidhi RastogiApril 14, 2025

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Apellis Pharmaceuticals Reports the US FDA’s sNDA Acceptance & Priority Review of Empaveli for C3 Glomerulopathy and Primary IC-MPGN

Shots:The US FDA has accepted sNDA & granted priority review to Empaveli for C3 glomerulopathy (C3G) & primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) pts (PDUFA: Jul 28, 2025)Submission was backed by P-III (VALIANT) trial assessing Empaveli (Q2W) vs PBO in C3G & primary IC-MPGN pts (≥12yrs., n=124) for 26wks. followed by 26wk. open-label…

ByRidhi RastogiApril 2, 2025

News

Sanofi Reports the US FDA’s Acceptance & Priority Review of Tolebrutinib for Non-Relapsing Secondary Progressive Multiple Sclerosis

Shots:The US FDA has accepted NDA & granted priority review to tolebrutinib for nrSPMS & slowing disability progression independent of relapse (PDUFA: Sep 28, 2025); MAA is under EMA reviewSubmission was backed by P-III trials, incl. HERCULES for nrSPMS & GEMINI 1 & 2 for relapsing MS, along with clinical & preclinical data…

ByRidhi RastogiMarch 25, 2025

News

The EC Approves BMS’ Opdivo + Yervoy as 1L Treatment for Hepatocellular Carcinoma

Shots:The EC has approved Opdivo + Yervoy as 1L therapy to treat inoperable or advanced HCC adult pts in 30 EEA states; sBLA under the US FDA’s priority review (PDUFA: Apr 21, 2025) Approval was based on P-III (CheckMate-9DW) trial assessing Opdivo (1mg/kg, Q3W) + Yervoy (3mg/kg, Q3W), followed by Opdivo alone (480mg,…

ByRidhi RastogiMarch 10, 2025

News

Regeneron and Sanofi Report the US FDA’s sBLA Acceptance for Dupixent to Treat Bullous Pemphigoid (BP)

Shots:The US FDA has accepted & granted priority review to the sBLA of Dupixent for the treatment of adults with BP (PDUFA: Jun 20, 2025) sBLA was supported by P-II/III (ADEPT) trial assessing Dupixent vs PBO in pts (n=106) for 52wks., that met its 1EP of 5x pts achieving sustained disease remission at…

ByRidhi RastogiFebruary 18, 2025

News

Johnson & Johnson Reports the US FDA’s BLA Acceptance of Nipocalimab with Priority Review for Generalized Myasthenia Gravis (gMG)

Shots:The US FDA has accepted & granted priority review to the BLA of nipocalimab for treating patients with antibody positive (anti-AChR, anti-MuSK, anti-LRP4) gMG, based on P-III (Vivacity-MG3) trial. MAA in this indication has been filed with the EMA on Sep 11, 2024 The P-III study assessed safety & efficacy of nipocalimab +…

ByRidhi RastogiJanuary 10, 2025

New Drug Designations – April 2025

BioCryst Reports the US FDA’s NDA Acceptance and Priority Review of Orladeyo for Hereditary Angioedema (HAE)

Cytokinetics Reports P-III (MAPLE-HCM) Trial Data of Aficamten for Obstructive Hypertrophic Cardiomyopathy

REGENXBIO Reports the US FDA’s BLA Acceptance and Priority Review of RGX-121 for Treating Mucopolysaccharidosis II

The US FDA Approves BMS’ Opdivo + Yervoy as 1L Treatment for Hepatocellular Carcinoma (HCC)

Apellis Pharmaceuticals Reports the US FDA’s sNDA Acceptance & Priority Review of Empaveli for C3 Glomerulopathy and Primary IC-MPGN

Sanofi Reports the US FDA’s Acceptance & Priority Review of Tolebrutinib for Non-Relapsing Secondary Progressive Multiple Sclerosis

The EC Approves BMS’ Opdivo + Yervoy as 1L Treatment for Hepatocellular Carcinoma

Regeneron and Sanofi Report the US FDA’s sBLA Acceptance for Dupixent to Treat Bullous Pemphigoid (BP)

Johnson & Johnson Reports the US FDA’s BLA Acceptance of Nipocalimab with Priority Review for Generalized Myasthenia Gravis (gMG)

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