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Regenxbio
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, 2025 As per Jan 2025 partnership, NS Pharma (Nippon Shinyaku’s subsidiary) will commercialize RGX-121 in the US post approval while REGENXBIO will handle…
May 14, 2025

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Apellis
NEWS

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…
April 2, 2025

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Sanofi
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 review Submission was backed by P-III trials, incl. HERCULES for nrSPMS & GEMINI 1 & 2 for relapsing MS, along with clinical & preclinical data…
March 25, 2025

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J&J
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 +…
January 10, 2025

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Hutchmed
NEWS

HUTCHMED Reports the NMPA’s NDA Acceptance of Orpathys Plus Tagrisso with Priority Review for EGFR-Mutated NSCLC with MET Amplification

Shots: The NMPA has accepted and granted priority review to NDA of Orpathys (savolitinib) + Tagrisso (osimertinib) for treating locally advanced or metastatic EGFR-mutated NSCLC with MET amplification in patients progressed on 1L EGFR inhibitor therapy. HUTCHMED will receive milestone from AstraZeneca Submission was based on P-III (SACHI) study assessing the safety & efficacy…
January 2, 2025

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New Drug Designations - October 2024
INSIGHTS+

New Drug Designations – October 2024

Shots:   PharmaShots' designation report provides a concise overview of the latest drug designations by major regulatory authorities, including the FDA, NMPA, and EMA   The October 2024 report covers designations granted to 33 drugs and 2 devices, encompassing 14 small molecules, 4 biologics, 8 cell and gene therapies, and 2 medical devices.  Significant trends this month…
November 26, 2024

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New Drug Designations - September 2024
INSIGHTS+

New Drug Designations – September 2024

Shots:   PharmaShots' designation report provides a concise overview of several drugs and their designations by the FDA, NMPA, Health Canada and EMA.  The September 2024 report covers regulatory designations for 42 drugs and 1 device, including 16 small molecules, 6 biologics, 12 cell and gene therapies, and 1 device, among others.  Significant trends this month…
October 29, 2024

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