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

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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

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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

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Bruno Vision Care Reports the US FDA Clearance of Deseyne for Presbyopia

Shots: FDA has cleared Deseyne, a daily disposable soft contact lens for presbyopia that uses patented Extended Depth of Focus technology Deseyne provides continuous near, intermediate, & distance vision through Cataltheia’s patented hyper-refractive central zone, showing a clinical advantage over multifocal lenses with minimal patient adaptation, strong comfort, & no adverse events reported in studies Made…

ByRidhi RastogiDecember 26, 2025

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Mabwell Receives the US FDA IND Clearance to Initiate P-IIa Trial of 9MW1911 in COPD

Shots: The US FDA has granted IND clearance to initiate a P-IIa trial of 9MW1911 for the treatment of patients with mod. to sev. chronic obstructive pulmonary disease (COPD) In the China P-IIa trial (n=80) vs PBO, 9MW1911 showed dose-proportional exposure & dose-dependent efficacy, with the RP2D (n=30) reducing mod. to sev. COPD exacerbations by…

ByRidhi RastogiDecember 26, 2025

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Omeros Receives the FDA Approval for Yartemlea (Narsoplimab-wuug) to Treat TA-TMA

Shots: The US FDA has approved Yartemlea for the treatment of HSCT-Associated Thrombotic Microangiopathy, with US launch planned for Jan 2026. MAA is under the EMA’s review, with a decision expected in mid-2026 Approval was based on the TA-TMA Study (n=28) plus EAP (N=221 adults & pediatric pts), where 19 pts (13 adult & 6…

ByRidhi RastogiDecember 26, 2025

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Agios Reports the US FDA Approval of Aqvesme for Anemia in Alpha or Beta Thalassemia

Shots: The US FDA has approved Agios’ Aqvesme (mitapivat) for the treatment of anemia in adults with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia Approval was based on the global P-III (ENERGIZE) and (ENERGIZE-T) studies (n=452), which met all 1EPs and 2EPs, demonstrating significant improvements in hemoglobin, fatigue, and reductions in transfusion burden vs PBO Aqvesme…

ByPrince GiriDecember 25, 2025

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SN BioScience Receives US FDA ODD for SNB-101 in Gastric Cancer

Shots: US FDA has granted Orphan Drug Designation (ODD) to SN BioScience’s SNB-101 for the treatment of gastric cancer, including gastroesophageal junction cancer SNB-101 is currently under P-Ib/II development for small cell lung cancer and is being evaluated across multiple solid tumor indications in Korea, Europe and US SNB-101 is a polymer nanoparticle formulation of…

ByPrince GiriDecember 25, 2025

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

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

Bruno Vision Care Reports the US FDA Clearance of Deseyne for Presbyopia

Mabwell Receives the US FDA IND Clearance to Initiate P-IIa Trial of 9MW1911 in COPD

Omeros Receives the FDA Approval for Yartemlea (Narsoplimab-wuug) to Treat TA-TMA

Agios Reports the US FDA Approval of Aqvesme for Anemia in Alpha or Beta Thalassemia

SN BioScience Receives US FDA ODD for SNB-101 in Gastric Cancer

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