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

NEWS

Daiichi Sankyo Reports the Health Canada’s Approval of Enhertu for HER2 Low/Ultralow Metastatic Breast Cancer (MBC)

Shots: Health Canada has approved Enhertu monotx. for adult with inoperable HR+, HER2-low/ultralow MBC who have received ≥1 endocrine therapy (ET) & are ineligible for ET as the next line of therapy Approval was based on the P-III (DESTINY-Breast06) trial (N=866) assessing Enhertu (5.4mg/kg) vs CT (capecitabine/paclitaxel/nab-paclitaxel) in HR+, HER2-low (n=713) & ultralow (n=152) pts Trial showed improved PFS…

ByRidhi RastogiOctober 31, 2025

NEWS

Merck Receives EC’s Approval for Perioperative Keytruda for Locally Advanced Head and Neck Squamous Cell Carcinoma (LA-HNSCC)

Shots: The EC has approved neoadj. Keytruda followed by adj. Keytruda + SoC radiotherapy (RT) ± cisplatin & then as a single agent to treat operable LA-HNSCC tumors expressing PD-L1 (CPS ≥1) across all 30 EEA states Approval was based on P-III (KEYNOTE-689) trial assessing neoadj. Keytruda (200mg, IV, Q3W × 2 cycles), followed by adj. Keytruda (15…

ByRidhi RastogiOctober 30, 2025

INSIGHTS+

New Drug Designations – September 2025   

Shots:    PharmaShots’ Designation Report provides a concise overview of the latest drug designations granted by major regulatory authorities, including the FDA, EMA, MHLW, Health Canada, and NMPA  The September 2025 report covers designations granted to 33 drugs and 4 medical devices, spanning 12 small molecules, 6 biologics, 8 cell and gene therapies & 4 medical devices among others  …

ByRidhi RastogiOctober 28, 2025

NEWS

AstraZeneca Reports the EC’s Approval of Koselugo (Selumetinib) for Treating Neurofibromatosis Type 1

Shots: The EC has approved Koselugo for the treatment of symptomatic, inoperable plexiform neurofibromas (PN) in adults with neurofibromatosis type 1 (NF1) Approval was based on the P-III (KOMET) study assessing Koselugo (PO) vs PBO in 145 adults with NF1 & symptomatic, inoperable PNs across 13 countries incl. North America, South America, Europe, Asia &…

ByRidhi RastogiOctober 28, 2025

NEWS

Syndax Pharmaceuticals’ Revuforj (Revumenib) Receives the US FDA’s Approval for NPM1-Mutated R/R Acute Myeloid Leukemia

Shots: The US FDA has approved label expansion of Revuforj to treat pts (≥1yrs.) with nucleophosmin 1 (NPM1)-mutated r/r acute myeloid leukemia (AML) who have no satisfactory alternative treatment options Approval was supported by P-II results from the P-I/II (AUGMENT-101) trial, which showed a 23% CR + CRh rate (15/65), median response time of 2.8mos.,…

ByRidhi RastogiOctober 28, 2025

NEWS

Eli Lilly’s Omvoh (mirikizumab-mrkz) Receives the US FDA’s Approval for Ulcerative Colitis

Shots: The US FDA has approved Omvoh (200mg/2mL, SC, QM) as a maintenance treatment of adults with mod. to sev. active ulcerative colitis; PFS & prefilled pen to be available in early 2026 Approval was based on P-I trial assessing Omvoh (200mg/2mL, SC) single injection vs 2 100mg/1mL injections in participants, which showed bioequivalence between the…

ByDipanshu DixitOctober 28, 2025

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

Daiichi Sankyo Reports the Health Canada’s Approval of Enhertu for HER2 Low/Ultralow Metastatic Breast Cancer (MBC)

Merck Receives EC’s Approval for Perioperative Keytruda for Locally Advanced Head and Neck Squamous Cell Carcinoma (LA-HNSCC)

New Drug Designations – September 2025

AstraZeneca Reports the EC’s Approval of Koselugo (Selumetinib) for Treating Neurofibromatosis Type 1

Syndax Pharmaceuticals’ Revuforj (Revumenib) Receives the US FDA’s Approval for NPM1-Mutated R/R Acute Myeloid Leukemia

Eli Lilly’s Omvoh (mirikizumab-mrkz) Receives the US FDA’s Approval for Ulcerative Colitis

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