Cogent Biosciences Reports US FDA’s NDA Submission of Bezuclastinib for NonAdvanced Systemic Mastocytosis

Shots: The US FDA has received the NDA of bezuclastinib for the treatment of NonAdvanced Systemic Mastocytosis (NonAdvSM) NDA was supported by the SUMMIT trial, which met its 1 & 2EPs, showing clinical benefit across all symptom domains, incl. 11 individual patient-reported symptoms as well as the most severe symptom, along with correlated biomarker reductions…

ByRidhi RastogiDecember 31, 2025

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Ultragenyx Completes FDA Rolling BLA Submission for DTX401 in Glycogen Storage Disease Type Ia (GSDIa)

Shots: Ultragenyx has completed rolling submission of a BLA to the FDA seeking approval of DTX401 (pariglasgene brecaparvovec) for the treatment of GSDIa BLA is supported by data from 52 pts with 6yrs. of follow-up. Previously reported P-III (GlucoGene) trial results showed significant reductions in both quantity & frequency of daily cornstarch intake, improved euglycemia…

ByRidhi RastogiDecember 31, 2025

NEWS

Vanda Pharmaceuticals Reports the US FDA Approval of Nereus (Tradipitant) to Prevent Vomiting Induced by Motion

Shots: The US FDA has granted Nereus for the prevention of vomiting induced by motion, backed by 3 trials, incl. 2 P-III real-world boat studies (Motion Syros & Motion Serifos) & 1 supporting study in pts with documented motion sickness In Motion Syros (n=365) & Motion Serifos (n=316), vomiting incidence was 18.3–19.5% & 10.4–18.3% vs 44.3%…

ByRidhi RastogiDecember 31, 2025

NEWS

HUTCHMED Reports NMPA’s NDA Acceptance with Priority Review of Savolitinib for Gastric Cancer Patients with MET Amplification

Shots: The Chinese NMPA has accepted NDA & granted priority review to savolitinib for the treatment of LA/M gastric cancer or gastroesophageal junction (GC/GEJ) adenocarcinoma pts with MET amplification who have failed ≥2 prior systemic treatments NDA was supported by the P-II trial of savolitinib in gastric cancer pts with MET amplification in China, which met its 1EP of improved ORR by…

ByRidhi RastogiDecember 30, 2025

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StimLabs Reports the US FDA 510(k) Clearance of Theracor for Acute and Chronic Wound Management

Shots: The FDA has granted 510(k) clearance to Theracor, a human umbilical cord-derived medical device offered in sheet form, for wound care Indicated for the same wound types as StimLabs’ Corplex P, Theracor is used for partial & full-thickness wounds, pressure, venous, diabetic, & chronic vascular ulcers, tunneled/undermined wounds, surgical sites, trauma-related injuries, & draining…

ByRidhi RastogiDecember 30, 2025

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INOVIO Reports the US FDA’s BLA Acceptance of INO-3107 for Recurrent Respiratory Papillomatosis (RRP)

Shots: The US FDA has accepted BLA of INO-3107 for the treatment for adults with RRP, granting PDUFA target action date of Oct 30, 2026 INOVIO filed a BLA for INO-3107 under the accelerated approval pathway & plans to engage with the FDA to align on requirements, while confirming it is not pursuing approval under…

ByRidhi RastogiDecember 30, 2025

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Praxis Precision Medicines Reports the US FDA Breakthrough Therapy Designation of Ulixacaltamide for Essential Tremor

Shots: The US FDA has granted BTD to ulixacaltamide for the treatment of patients with essential tremor (ET) Designation was based on the topline data from the Essential3 program, consisting of Study 1 & 2, where in Study 1, ulixacaltamide improved mADL11 (1EP) by 4.3 points at Wk. 8, while Study 2 showed superior maintenance of…

ByRidhi RastogiDecember 30, 2025

NEWS

Mereo BioPharma Reports P-III (ORBIT & COSMIC) Trials Findings on Setrusumab in Osteogenesis Imperfecta

Shots: Mereo BioPharma has reported setrusumab (UX143) results from the global P-III (ORBIT) trial vs PBO & P-III (COSMIC) trial vs bisphosphonates (IV) in 159 pts (5-25yrs.) & 69 pts (2 to <7 yrs.), respectively, with osteogenesis imperfecta Neither study met its 1EP of reduced annualized clinical fracture rate, but both showed statistically significant improvements…

ByRidhi RastogiDecember 30, 2025

NEWS

ARS Pharmaceuticals Reports NMPA Approval of neffy 2mg (Epinephrine Nasal Spray) to Treat Type I Allergic Reactions

Shots: The Chinese NMPA has approved neffy 2mg for the emergency treatment of type I allergic reactions (anaphylaxis) in adults & children (≥30kg), with availability anticipated in the spring of 2026. Filing for neffy 1mg for children (15 to <30kg) to the NMPA is expected in the coming mos. In 2021, ARS Pharma licensed neffy…

ByRidhi RastogiDecember 30, 2025

NEWS

Adlai Nortye Licenses AN9025 to ASK Pharm in a ~$230M Deal

Shots: Adlai Nortye has granted ASK Pharm exclusive rights to develop, manufacture, & commercialize AN9025 in mainland China, Hong Kong & Macao, with Adlai retaining rights in other regions As per the deal, Adlai will receive ~$230M, incl. >$20M in upfront & near-term milestone, plus tiered royalties ranging from a high single-digit to mid-teens %…

ByRidhi RastogiDecember 29, 2025

Ridhi Rastogi

Cogent Biosciences Reports US FDA’s NDA Submission of Bezuclastinib for NonAdvanced Systemic Mastocytosis

Ultragenyx Completes FDA Rolling BLA Submission for DTX401 in Glycogen Storage Disease Type Ia (GSDIa)

Vanda Pharmaceuticals Reports the US FDA Approval of Nereus (Tradipitant) to Prevent Vomiting Induced by Motion

HUTCHMED Reports NMPA’s NDA Acceptance with Priority Review of Savolitinib for Gastric Cancer Patients with MET Amplification

StimLabs Reports the US FDA 510(k) Clearance of Theracor for Acute and Chronic Wound Management

INOVIO Reports the US FDA’s BLA Acceptance of INO-3107 for Recurrent Respiratory Papillomatosis (RRP)

Praxis Precision Medicines Reports the US FDA Breakthrough Therapy Designation of Ulixacaltamide for Essential Tremor

Mereo BioPharma Reports P-III (ORBIT & COSMIC) Trials Findings on Setrusumab in Osteogenesis Imperfecta

ARS Pharmaceuticals Reports NMPA Approval of neffy 2mg (Epinephrine Nasal Spray) to Treat Type I Allergic Reactions

Adlai Nortye Licenses AN9025 to ASK Pharm in a ~$230M Deal

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