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INSIGHTS+

New Drug Designations: June 2025 

Shots:      PharmaShots’ Designation Report offers a concise overview of the latest drug and device designations granted by major regulatory authorities, including the FDA, EMA, MHLW, Health Canada, and NMPA   The June 2025 edition covers designations awarded to 43 drugs and 3 medical devices, comprising 17 small molecules, 7 biologics, 8 cell and gene therapies, and…
July 29, 2025

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NEWS

Deciphera Pharmaceuticals Reports the CHMP’s Positive Opinion of Romvimza for Symptomatic Tenosynovial Giant Cell Tumor (TGCT)

Shots: The CHMP has recommended Romvimza (vimseltinib) to treat symptomatic TGCT with physical function deterioration in adults for which surgery may lead to functional impairment or severe morbidity; EC’s decision is expected in Q3'25 Opinion was based on P-I/II trial as well as P-III (MOTION) trial, which evaluated Romvimza vs PBO in surgery-ineligible pts without…
July 29, 2025

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NEWS

BI Collaborates with Re-Vana Therapeutics to Develop Long-Acting Therapeutics for Eye Diseases

Shots: BI & Re-Vana Therapeutics have entered into a strategic collaboration & license agreement to develop extended-release therapies for eye diseases As per the deal, BI will obtain target exclusivity, while Re-Vana will receive over $1B in total potential deal value for the initial 3 targets, incl. upfront, development, regulatory, & commercial milestones, plus royalties…
July 29, 2025

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NEWS

PTC Therapeutics Receives the US FDA’s Approval for Sephience to Treat Phenylketonuria

Shots: The US FDA has approved Sephience (sepiapterin) for the treatment of phenylketonuria (PKU) in pts of all ages, with broad labeling for hyperphenylalaninemia in adults & pediatric pts (≥1mos.) with sepiapterin-responsive PKU; review is ongoing in Japan & Brazil Approval was based on APHENITY study results & long-term extension data showing durable effects and…
July 29, 2025

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Ionis
NEWS

Ionis Reports the CHMP’s Positive Opinion on Tryngolza for Familial Chylomicronemia Syndrome

Shots: The CHMP has recommended Tryngolza (olezarsen) as an adjunct to diet in adult patients for the treatment of genetically confirmed familial chylomicronemia syndrome; EC’s decision is expected in Q4’25 Opinion was based on P-III (Balance) trial assessing (Q4W) vs PBO, which showed reduced triglyceride levels at 6mos., sustained through 12mos., with decrease in acute…
July 29, 2025

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NEWS

Sobi Reports the US FDA’s Approval of Doptelet (Avatrombopag) to Treat Pediatric Immune Thrombocytopenia

Shots: The US FDA has approved Doptelet to treat thrombocytopenia in pts (≥1yrs.) with persistent or chronic ITP who have not responded to prior treatments. Approval also incl. a new formulation, Doptelet Sprinkle (avatrombopag) oral granules, for children (1-6yrs.) Approval was based on P-III (AVA-PED-301) study assessing Doptelet in pediatric ITP pts, which showed that…
July 28, 2025

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NEWS

Memo Therapeutics Reports P-II (SAFE KIDNEY II) Trial Data on Potravitug to Treat BKPyV Infection in Kidney Transplant Recipients

Shots: Memo Therapeutics has reported P-II (SAFE KIDNEY II) trial data assessing potravitug vs PBO for the treatment of BK polyomavirus (BKPyV) infection in kidney transplant recipients (n=95) Trial didn’t meet its 1EP of undetectable BKPyV DNAemia but showed superior viral response (≥1-log₁₀ reduction) & resolution of biopsy-proven BKPyVAN by Wk. 20, with histological improvement from…
July 28, 2025

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