Johnson & Johnson’s Darzalex Faspro Secures the US FDA’s Approval for High-Risk Smouldering Multiple Myeloma (SMM)

Shots: FDA has approved Darzalex Faspro (daratumumab & hyaluronidase-fihj) for high‑risk SMM based on the P-III (AQUILA) study assessing Darzalex Faspro alone vs active monitoring in adults, which met its 1EP of improved PFS by 51%, with 63.1% vs 40.7% pts progression-free at a mFU of 65.2mos. Trial also showed an improved ORR (63.4% vs…

ByRidhi RastogiNovember 7, 2025

NEWS

argenx Receives Health Canada’s Approval for Vyvgart SC to Treat Chronic Inflammatory Demyelinating Polyneuropathy

Shots: Health Canada has approved Vyvgart (efgartigimod alfa; QW, SC, PFS) as a monotx. for adults with chronic inflammatory demyelinating polyneuropathy (CIDP) Approval was based on the ADHERE trial, showing 69% (221/322) of Vyvgart SC-treated pts had clinical improvement, regardless of prior treatment. The study met its 1EP with a 61% reduction in relapse risk vs…

ByRidhi RastogiNovember 6, 2025

NEWS

Neurona Therapeutics’ NRTX-1001 Secures the EMA’s Priority Medicines (PRIME) Designation for Drug-Resistant Focal Epilepsy

Shots: The EMA has granted PRIME designation to NRTX-1001 for adults with drug-resistant focal epilepsy, based on preclinical & emerging clinical data NRTX-1001 is also being evaluated in 2 ongoing P-I/II trials for drug-resistant unilateral & bilateral mesial temporal lobe epilepsy (MTLE), with P-III (EPIC) trial planned to begin in H2’25 for drug-resistant MTLE The…

ByRidhi RastogiNovember 6, 2025

NEWS

Averoa Receives the MHRA’s Approval for Xoanacyl to Treat Chronic Kidney Disease (CKD)

Shots: The UK’s MHRA has approved Xoanacyl (ferric citrate complex) for the treatment of concomitant elevated serum phosphorus & iron deficiency in CKD adults via International Recognition Procedure (Route B), following EC’s approval in Jun 2025 In Dec 2022, Averoa obtained an exclusive license from Akebia Therapeutics to develop & commercialize Xoanacyl in the EEA,…

ByRidhi RastogiNovember 6, 2025

NEWS

Meitheal Pharmaceuticals Receives the US FDA Approval for Contepo (Fosfomycin) Injection to Treat cUTI in Adults

Shots: Meitheal Pharmaceuticals received the US FDA approval for Contepo (fosfomycin) for injection to treat complicated urinary tract infections (cUTI), including acute pyelonephritis in adults caused by E. coli and K. pneumoniae The approval was supported by the P-II/III ZEUS (ZTI-01) study that assessed the safety and efficacy of the treatment on hospitalized pts and…

ByPrince GiriNovember 5, 2025

NEWS

UCB Reports the US FDA’s Approval of Kygevvi (Doxecitine + Doxribtimine) for Thymidine Kinase 2 Deficiency (TK2d)

Shots: FDA has approved Kygevvi (2g/2g; PO) for adults & pediatric pts with TK2d whose symptom onset occurred ≤12yrs. of age. Kygevvi is expected to launch in the US by Q1’26, & is under EMA's review, with additional regulatory filings planned Approval was backed by a P-II trial, 2 retrospective chart review studies, & an…

ByRidhi RastogiNovember 4, 2025

NEWS

Calluna Pharma’s CAL101 Receives the US FDA’s Orphan Drug Designation to Treat Idiopathic Pulmonary Fibrosis (IPF)

Shots: The US FDA has granted ODD to CAL101 for the treatment of idiopathic pulmonary fibrosis (IPF) CAL101 (IV, QM for 7mos.) is being investigated in a P-II (AURORA) trial against PBO in 150 IPF pts, with enrolment open in the sites across US, UK, EU, Turkey, & South Korea, evaluating lung function per forced vital…

ByRidhi RastogiNovember 3, 2025

NEWS

Belite Bio Reports the MHRA’s CMA Application Acceptance for Tinlarebant to Treat Stargardt Disease

Shots: The UK’s MHRA has accepted a conditional marketing authorization (CMA) application for Tinlarebant to treat Stargardt disease CMA was based on the interim results from P-III (DRAGON) trial assessing Tinlarebant vs PBO in 104 adolescents with Stargardt disease across 11 jurisdictions, incl. the US, UK, Germany, France, Belgium, Switzerland, Netherlands, China, Hong Kong, Taiwan,…

ByRidhi RastogiNovember 3, 2025

WEEKLY SNAPSHOT

PharmaShots Weekly Snapshots (Oct 27, 2025 – Oct 31, 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: Roche Reports P-III (INShore) Trial Findings on Gazyva (Obinutuzumab) for Idiopathic Nephrotic Syndrome Read More: Roche Merck Reports the P-III (LITESPARK-022) Trial Data on Welireg + Keytruda to Treat Clear…

ByRidhi RastogiOctober 31, 2025

NEWS

BioMarin Reports the US FDA’s sBLA Acceptance & Priority Review of Palynziq (Pegvaliase-pqpz) to Treat Phenylketonuria

Shots: The US FDA has accepted sBLA & granted priority review to Palynziq for the treatment of adolescents (12-17yrs.) with phenylketonuria (PDUFA: Feb 28, 2026) sBLA was supported by the P-III (PEGASUS) trial comparing Palynziq to diet alone in 55 adolescents with phenylketonuria, which showed reduced blood phenylalanine (Phe); results were presented at ICIEM’25 Palynziq…

ByRidhi RastogiOctober 31, 2025

Johnson & Johnson’s Darzalex Faspro Secures the US FDA’s Approval for High-Risk Smouldering Multiple Myeloma (SMM)

argenx Receives Health Canada’s Approval for Vyvgart SC to Treat Chronic Inflammatory Demyelinating Polyneuropathy

Neurona Therapeutics’ NRTX-1001 Secures the EMA’s Priority Medicines (PRIME) Designation for Drug-Resistant Focal Epilepsy

Averoa Receives the MHRA’s Approval for Xoanacyl to Treat Chronic Kidney Disease (CKD)

Meitheal Pharmaceuticals Receives the US FDA Approval for Contepo (Fosfomycin) Injection to Treat cUTI in Adults

UCB Reports the US FDA’s Approval of Kygevvi (Doxecitine + Doxribtimine) for Thymidine Kinase 2 Deficiency (TK2d)

Calluna Pharma’s CAL101 Receives the US FDA’s Orphan Drug Designation to Treat Idiopathic Pulmonary Fibrosis (IPF)

Belite Bio Reports the MHRA’s CMA Application Acceptance for Tinlarebant to Treat Stargardt Disease

PharmaShots Weekly Snapshots (Oct 27, 2025 – Oct 31, 2025)

BioMarin Reports the US FDA’s sBLA Acceptance & Priority Review of Palynziq (Pegvaliase-pqpz) to Treat Phenylketonuria

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