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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, 20250Comments
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, 20250Comments
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, 20250Comments
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, 20250Comments
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, 20250Comments
VIEWPOINTS
Unlocking Approval: John Hayslip from Verastem Oncology in an Illuminating Conversation with PharmaShots 
Shots:  Did you know that more than 80 % of Low-grade Serous Ovarian Cancer (LGSOC) patients experience recurrence?  The US FDA recently approved Verastem Oncology’s Avmapki Fakzynja Co-Pack, as the first ever treatment of patients with KRAS-mutated recurrent LGSOC  In an illuminating dialogue exchange with PharmaShots, John Hayslip, CMO at Verastem Oncology, provides a detailed overview…
July 28, 20250Comments