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NEWS

KalVista Pharmaceuticals Reports EC and Swissmedic Approval of Ekterly (Sebetralstat) for Hereditary Angioedema (HAE) 

Shots:  The EC and Swissmedic have approved Ekterly to treat acute HAE attacks in pts (age≥12) across EEA states. Launch is expected in Germany in Q4’25 and in Switzerland in H2’26  Ekterly’s approval by the EC and Swissmedic was based on the P-III (KONFIDENT) trial. Published in NEJM (May 2024), results showed significantly faster symptom…
September 19, 2025

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NEWS

The US FDA Grants Accelerated Approval to Stealth BioTherapeutics’ Forzinity for Barth Syndrome 

Shots:  The US FDA has received accelerated approval from the US FDA for Forzinity (elamipretide HCl)] to improve muscle strength in pts with Barth syndrome (Wt.≥30 kg or 66 lbs)   Approval was based on efficacy and safety data from the TAZPOWER trial, which showed improved knee muscle strength  Forzinity is a mitochondrial cardiolipin binder. It…
September 19, 2025

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NEWS

The EC Approves Servier’s Voranigo (vorasidenib) for Grade 2 IDH-Mutant Glioma in the EU 

Shots:  The EC has approved Voranigo for treating patients (age: ≥12yrs.; wt≥ 40kg) with Gr2 astrocytoma or oligodendroglioma with a susceptible IDH1/2 mutation post-surgery, but do not currently require radiotherapy or CT across EEA states   Approval was based on the global pivotal P-III (INDIGO) trial, which demonstrated that vorasidenib significantly improved PFS and TTNI vs…
September 19, 2025

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Incyte
NEWS

Incyte Reports the US FDA’s sNDA Approval of Opzelura (Ruxolitinib) for Atopic Dermatitis (AD)

Shots: The FDA has approved Opzelura cream 1.5% for short-term and non-continuous treatment of mild to moderate AD in non-immunocompromised children (aged≥ 2), when other topical therapies are ineffective or unsuitable sNDA approval was based on the P-III (TRuE-AD3) trial which evaluated the safety and efficacy of ruxolitinib in over 300 children receiving 0.75%, 1.5%,…
September 19, 2025

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NEWS

Deciphera Pharmaceuticals Reports the EC’s Approval of Romvimza for Symptomatic Tenosynovial Giant Cell Tumor (TGCT)

Shots: The EC has approved Romvimza (vimseltinib) to treat symptomatic TGCT with physical function deterioration in adults for which surgery may lead to functional impairment or severe morbidity Approval was based on P-I/II trial as well as P-III (MOTION) trial, which evaluated Romvimza vs PBO in surgery-ineligible pts without prior anti-CSF1/CSF1R therapy (prior imatinib/nilotinib allowed)…
September 18, 2025

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WEEKLY SNAPSHOT

PharmaShots Weekly Snapshots (Sep 08, 2025 – Sep 12, 2025)   

This week, PharmaShots’ news was all about the updates on Clinical Trials, Regulatory, Pharma, MedTech, M&A, Biosimilar, and DigiHealth. Check out our full report below:      AstraZeneca Reports P-III (FLAURA2) Trial Findings on Tagrisso Regimen to Treat EGFRm NSCLC  Read More: AstraZeneca  Eli Lilly Reports P-III (BRUIN CLL-313) Trial Data on Jaypirca for 1L…
September 12, 2025

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Ionis
NEWS

Ionis’ ION582 Receives the US FDA’s Breakthrough Therapy Designation to Treat Angelman Syndrome

Shots: The US FDA has granted BTD to ION582 (UBE3A protein expression stimulant) for the treatment of Angelman syndrome Designation was based on P-I/II (HALOS) trial assessing ION582 in pts with Angelman syndrome, which showed improvements across all functional domains incl. communication, cognition & motor function; safety was favorable Additionally, ION582 is being investigated in…
September 12, 2025

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NEWS

Ferring Reports the PMDA’s NDA Acceptance of Nadofaragene Firadenovec to Treat BCG-Unresponsive High-Risk Non-Muscle-Invasive Bladder Cancer (HR-NMIBC)

Shots: Japan’s PMDA has accepted the NDA of nadofaragene firadenovec (intravesically; Q3W) for the treatment of Bacillus Calmette-Guérin (BCG)-unresponsive HR-NMIBC with CIS ± papillary tumors (Ta/T1) NDA was backed by P-III study evaluating nadofaragene in 157 pts, where 5yr. follow-up data showed OS rate of 80% & a 49% cystectomy-free survival in adults with CIS…
September 12, 2025

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