BPGbio’s BPM31510 Receives the US FDA Orphan Drug Designation to Treat Primary CoQ10 Deficiency

Shots: The US FDA has granted ODD to BPM31510, for the treatment of primary coenzyme Q10 (CoQ10) deficiency (PCQD) Designation was supported by insights from BPGbio’s NAi Interrogative Biology platform, which leverages Bayesian AI to analyze & map thousands of human biological data points, validating BPM31510’s therapeutic potential BPM31510 is designed for IV administration of CoQ10 directly…

ByRidhi RastogiJanuary 23, 2026

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SN BioScience Receives US FDA ODD for SNB-101 in Gastric Cancer

Shots: US FDA has granted Orphan Drug Designation (ODD) to SN BioScience’s SNB-101 for the treatment of gastric cancer, including gastroesophageal junction cancer SNB-101 is currently under P-Ib/II development for small cell lung cancer and is being evaluated across multiple solid tumor indications in Korea, Europe and US SNB-101 is a polymer nanoparticle formulation of…

ByPrince GiriDecember 25, 2025

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GSK’s Risvutatug Rezetecan Secures the US FDA’s Orphan Drug Designation for Treating SCLC

Shots: The US FDA has granted ODD to GSK’s risvutatug rezetecan (GSK5764227) for the treatment of small-cell lung cancer (SCLC) Designation was supported by preliminary P-I (ARTEMIS-001) trial data showing durable responses in pts with extensive-stage SCLC treated with risvutatug rezetecan GSK’227 is a B7-H3-targeted ADC developed by Hansoh Pharma for various solid tumors. GSK…

ByRidhi RastogiDecember 11, 2025

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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

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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

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Corcept Therapeutics Reports the EMA’s MAA Submission of Relacorilant to Treat Platinum-Resistant Ovarian Cancer

Shots: The EMA has received MAA of relacorilant for the treatment of Pt-resistant ovarian cancer MAA was supported by P-III (ROSELLA) & P-II trials, where relacorilant + nab-paclitaxel demonstrated improved PFS & OS compared to nab-paclitaxel monotx., with no need for biomarker selection & showed favorable safety Relacorilant (PO) is a GR antagonist that is…

ByRidhi RastogiOctober 15, 2025

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Corcept Therapeutics Reports the US FDA’s NDA Acceptance of Relacorilant to Treat Platinum-Resistant Ovarian Cancer

Shots: The US FDA has accepted NDA of relacorilant for the treatment of Pt-resistant ovarian cancer (PDUFA: Jul 11, 2026) NDA was supported by P-III (ROSELLA) & P-II trials, where relacorilant + nab-paclitaxel demonstrated improved PFS & OS compared to nab-paclitaxel monotx., with no need for biomarker selection & favorable safety Additionally, relacorilant (GR antagonist)…

ByRidhi RastogiSeptember 11, 2025

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Pulmovant and Roivant’s Mosliciguat Receives MHLW’s Orphan Drug Designation for Pulmonary Hypertension Associated with Interstitial Lung Disease

Shots: Japan’s MHLW has granted ODD to mosliciguat for the treatment of pulmonary hypertension associated with interstitial lung disease (PH-ILD) Mosliciguat (dry powder inhaler) is being evaluated in an ongoing global P-II (PHocus) trial against PBO among ~120 PH-ILD adults, with trial design to be presented at ERS Congress 2025 Mosliciguat is an sGC activator that…

ByRidhi RastogiSeptember 5, 2025

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New Drug Designations: July 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 July 2025 edition covers designations awarded to 47 drugs and 3 medical devices, comprising 23 small molecules, 9 biologics, 8 cell and gene therapies, and…

ByRidhi RastogiAugust 26, 2025

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Sanofi’s Rilzabrutinib Secures the EMA’s Orphan Drug Designation to Treat IgG4-Related Disease

Shots: The EMA has granted ODD to rilzabrutinib for the treatment of IgG4-related disease (IgG4-RD) Rilzabrutinib (PO) is being evaluated in a P-II trial for IgG4-RD with glucocorticoids vs glucocorticoids alone, showing reduced flares, improved disease markers, & steroid sparing over 52 weeks; data presented at EULAR 2025 Rilzabrutinib, a reversible covalent BTK inhibitor, is…

ByRidhi RastogiAugust 14, 2025

BPGbio’s BPM31510 Receives the US FDA Orphan Drug Designation to Treat Primary CoQ10 Deficiency

SN BioScience Receives US FDA ODD for SNB-101 in Gastric Cancer

GSK’s Risvutatug Rezetecan Secures the US FDA’s Orphan Drug Designation for Treating SCLC

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

New Drug Designations – September 2025

Corcept Therapeutics Reports the EMA’s MAA Submission of Relacorilant to Treat Platinum-Resistant Ovarian Cancer

Corcept Therapeutics Reports the US FDA’s NDA Acceptance of Relacorilant to Treat Platinum-Resistant Ovarian Cancer

Pulmovant and Roivant’s Mosliciguat Receives MHLW’s Orphan Drug Designation for Pulmonary Hypertension Associated with Interstitial Lung Disease

New Drug Designations: July 2025

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