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NEWS

Neurona Therapeutics’ NRTX-1001 Secures the EMA’s Priority Medicines (PRIME) Designation for Drug-Resistant Focal Epilepsy

Shots: The EMA has granted PRIME designation to NRTX-1001 for adults with drug-resistant focal epilepsy, based on preclinical & emerging clinical data NRTX-1001 is also being evaluated in 2 ongoing P-I/II trials for drug-resistant unilateral & bilateral mesial temporal lobe epilepsy (MTLE), with P-III (EPIC) trial planned to begin in H2’25 for drug-resistant MTLE The…
November 6, 2025

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NEWS

BioMarin Reports the US FDA’s sBLA Acceptance & Priority Review of Palynziq (Pegvaliase-pqpz) to Treat Phenylketonuria

Shots: The US FDA has accepted sBLA & granted priority review to Palynziq for the treatment of adolescents (12-17yrs.) with phenylketonuria (PDUFA: Feb 28, 2026) sBLA was supported by the P-III (PEGASUS) trial comparing Palynziq to diet alone in 55 adolescents with phenylketonuria, which showed reduced blood phenylalanine (Phe); results were presented at ICIEM’25 Palynziq…
October 31, 2025

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INSIGHTS+

New Drug Designations – September 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 September 2025 report covers designations granted to 33 drugs and 4 medical devices, spanning 12 small molecules, 6 biologics, 8 cell and gene therapies & 4 medical devices among others  …
October 28, 2025

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NEWS

GSK’s Shingrix PFS Presentation Receives the CHMP’s Positive Opinion

Shots: The CHMP has recommended Shingrix in a PFS (Recombinant Zoster Vaccine or RZV), simplifying administration by eliminating vial reconstitution for the prevention of shingles (herpes zoster). EU marketing authorization is expected in December 2025 The opinion was based on data showing that the PFS matches the existing vaccine Shingrix combines glycoprotein E antigen with…
October 23, 2025

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Sanofi
NEWS

Sanofi’s Wayrilz (Rilzabrutinib) Receives the CHMP’s Positive Opinion to Treat Immune Thrombocytopenia (ITP)

Shots: The CHMP has recommended Wayrilz (rilzabrutinib) for adults with ITP unresponsive to other treatments, with a final decision expected soon; under regulatory review for ITP in China Opinion was based on the P-III (LUNA 3) trial assessing Wayrilz (400mg, BID, PO) vs PBO during a 12–24-wk, followed by 28 wks, and then a four-week…
October 22, 2025

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NEWS

Insmed’s Brinsupri (Brensocatib) Receives the CHMP’s Positive Opinion to Treat Non-Cystic Fibrosis Bronchiectasis (NCFB)

Shots: The CHMP has recommended Brinsupri (Brensocatib) for treating NCFB pts (≥12yrs.) with two or more exacerbations in the prior 12 mos. Opinion was based on P-III (ASPEN) trial (n=1721) & P-II (WILLOW) trial (n=256) assessing Brinsupri (10 or 25mg, PO) vs PBO in NCFB pts for 52 & 24wks., respectively, with WILLOW enrolling pts who had…
October 21, 2025

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Regeneron New
NEWS

Regeneron Reports the CHMP’s Positive Opinion for Adjuvant Libtayo to Treat High-Risk Cutaneous Squamous Cell Carcinoma (CSCC)

Shots: The CHMP has recommended Libtayo (cemiplimab) as an adj. treatment for CSCC adults who are at high risk for recurrence after surgery & radiation Opinion was based on the P-III (C-POST) trial assessing Libtayo adj. (350mg, IV, Q3W for first 12wks. followed by 700mg, IV, Q6W for 36wks.; n=209) vs PBO (n=206) to treat…
October 21, 2025

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NEWS

Corcept Therapeutics Reports the EMA’s MAA Submission of Relacorilant to Treat Platinum-Resistant Ovarian Cancer

Shots: The EMA has received MAA of relacorilant for the treatment of Pt-resistant ovarian cancer MAA was supported by P-III (ROSELLA) & P-II trials, where relacorilant + nab-paclitaxel demonstrated improved PFS & OS compared to nab-paclitaxel monotx., with no need for biomarker selection & showed favorable safety Relacorilant (PO) is a GR antagonist that is…
October 15, 2025

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