The CHMP Recommends Ionis and Otsuka’s Dawnzera (Donidalorsen) as a Prophylactic Treatment of Hereditary Angioedema (HAE)

Shots: The CHMP has recommended Dawnzera for the routine prevention of HAE attacks in pts (≥12yrs.) following the US FDA approval in Aug 2025; EC’s decision is expected in Q1’26 Opinion was based on P-III (OASIS-HAE) trial & OASISplus study, showing improvements across multiple domains, incl. sustained reduction in mean monthly HAE attack rate even when Dawnzera…

ByRidhi RastogiNovember 17, 2025

NEWS

Sanofi’s Teizeild (Teplizumab) Receives the CHMP’s Positive Opinion to Delay Progression of Type 1 Diabetes

Shots: The CHMP has recommended Teizeild to delay the onset of stage 3 type 1 diabetes (T1D) in adult & pediatric pts (≥8yrs.) with stage 2 T1D; regulatory review is ongoing in the other regions Opinion was based on the P-II (TN-10) trial assessing Teizeild (QD, n=44) vs PBO (n=32) for 14 days in 76…

ByRidhi RastogiNovember 17, 2025

WEEKLY SNAPSHOT

PharmaShots Weekly Snapshots (Nov 10, 2025 – Nov 14, 2025)    

This week, PharmaShots’ news was all about the updates on Clinical Trials, Regulatory, Pharma, MedTech, M&A, Animal Health, and Biosimilars. Check out our full report below:    Ionis Highlights P-III (CORE & CORE2) Trials Results of Olezarsen for Severe Hypertriglyceridemia (sHTG) at AHA 2025 Read More: Ionis AstraZeneca Reports P-III (NATRON) Trial Data on Fasenra (Benralizumab) to Treat Hypereosinophilic Syndrome (HES) …

ByRidhi RastogiNovember 14, 2025

NEWS

Lundbeck Reports the MHLW’s NDA Acceptance of Vyepti (Eptinezumab) for the Preventive Treatment of Migraine

Shots: The Japanese MHLW has accepted an NDA of Vyepti for the treatment of pts living with migraine, who are eligible for preventive therapy; regulatory review is also ongoing in China & South Korea The NDA was supported by extensive clinical data, incl. the P-III (SUNRISE) trial of Vyepti (100 or 300mg, IV) vs PBO…

ByRidhi RastogiNovember 14, 2025

NEWS

Outlook Therapeutics Reports the US FDA’s BLA Acceptance of ONS-5010 to Treat Wet Age-Related Macular Degeneration (wAMD)

Shots: The US FDA has accepted the resubmitted BLA of ONS-5010 (bevacizumab-vikg) for the treatment of wAMD, considering a Class 1 review & granting a PDUFA goal date Dec 31, 2025; if approved, it will be branded Lytenava ONS-5010 (intravitreal) is an ophthalmic bevacizumab formulation that binds all VEGF isoforms to block VEGF interaction with…

ByRidhi RastogiNovember 14, 2025

NEWS

Kura Oncology and Kyowa Kirin Report the US FDA’s Approval Komzifti (Ziftomenib) for NPM1-Mutated Acute Myeloid Leukemia (AML)

Shots: The US FDA has granted full approval to Komzifti (QD, PO) to treat adults with r/r AML with NPM1 mutation who have no satisfactory alternative treatment options before the PDUFA date of Nov 30, 2025 Approval was backed by P-I/II (KOMET-001) trial in 112 r/r NPM1-mutant AML pts, showing 21.4% CR + CRh with a median…

ByRidhi RastogiNovember 14, 2025

NEWS

Eisai and BioArctic Report the MHRA Approval of Leqembi (Lecanemab) as a Maintenance Therapy to Treat Early Alzheimer’s Disease

Shots: The UK’s MHRA has approved Leqembi (IV, Q4W) as a maintenance therapy for early Alzheimer’s pts after completing 18mos. of Q2W Leqembi treatment Leqembi is approved in 51 countries for early AD & under review in 9, while the Q4W maintenance is approved in the UK, China, the US, & others, with 4 applications…

ByRidhi RastogiNovember 14, 2025

NEWS

ITM Reports the US FDA’s NDA Acceptance for ¹⁷⁷Lu-edotreotide to Treat Gastroenteropancreatic Neuroendocrine Tumors (GEP-NETs)

Shots: The US FDA has accepted an NDA of 177Lu-edotreotide (ITM-11) for the treatment of GEP-NETs, with a PDUFA goal date of Aug 28, 2026 NDA was supported by the P-III (COMPETE) trial assessing ITM-11 vs everolimus (N=309) as a 1/2L treatment of inoperable, progressive, SSTR+ Grade 1 or 2 GEP-NETs, which met its 1EP of…

ByRidhi RastogiNovember 13, 2025

NEWS

BioNet’s VacPertagen Receives the CHMP’s Positive Opinion for Pertussis Disease

Shots: The CHMP has recommended VacPertagen as a booster aganist Pertussis for pts (age≥12) and for passive infant protection via maternal immunization during pregnancy VacPertagen is an acellular pertussis vaccine with two components, including recombinant Pertussis Toxin (PTgen) and filamentous haemagglutinin (FHA), providing targeted protection without unnecessary antigens VacPertagen’s benefits have been demonstrated in three clinical studies involving adults, adolescents, and pregnant women Ref: BioNet| Image: BioNet| Press Release…

ByDipanshu DixitNovember 13, 2025

NEWS

Fondazione Telethon’s Waskyra Receives the CHMP’s Positive Opinion for WAS

Shots: The CHMP has recommended Waskyra, an ex vivo gene therapy for Wiskott-Aldrich Syndrome (WAS) Waskyra is a single-administration therapy that uses a patient’s CD34+ hematopoietic stem and progenitor cells, modified with a lentiviral vector carrying the WAS gene The therapy will be available to pts at IRCCS Ospedale San Raffaele, where the clinical trial was conducted. The BLA for WAS…

ByDipanshu DixitNovember 13, 2025

The CHMP Recommends Ionis and Otsuka’s Dawnzera (Donidalorsen) as a Prophylactic Treatment of Hereditary Angioedema (HAE)

Sanofi’s Teizeild (Teplizumab) Receives the CHMP’s Positive Opinion to Delay Progression of Type 1 Diabetes

PharmaShots Weekly Snapshots (Nov 10, 2025 – Nov 14, 2025)

Lundbeck Reports the MHLW’s NDA Acceptance of Vyepti (Eptinezumab) for the Preventive Treatment of Migraine

Outlook Therapeutics Reports the US FDA’s BLA Acceptance of ONS-5010 to Treat Wet Age-Related Macular Degeneration (wAMD)

Kura Oncology and Kyowa Kirin Report the US FDA’s Approval Komzifti (Ziftomenib) for NPM1-Mutated Acute Myeloid Leukemia (AML)

Eisai and BioArctic Report the MHRA Approval of Leqembi (Lecanemab) as a Maintenance Therapy to Treat Early Alzheimer’s Disease

ITM Reports the US FDA’s NDA Acceptance for ¹⁷⁷Lu-edotreotide to Treat Gastroenteropancreatic Neuroendocrine Tumors (GEP-NETs)

BioNet’s VacPertagen Receives the CHMP’s Positive Opinion for Pertussis Disease

Fondazione Telethon’s Waskyra Receives the CHMP’s Positive Opinion for WAS

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