Ionis Highlights P-III (CORE & CORE2) Trials Results of Olezarsen for Severe Hypertriglyceridemia (sHTG) at AHA 2025

Shots: The P-III (CORE: n=617 & CORE2: n=446) trials assessed olezarsen (50 or 80mg, SC, Q4W) vs PBO for 12mos. in sHTG adults; the US FDA’s sNDA filing is expected by 2025 end Both trials met their 1EP with ~72% PBO-adjusted fasting triglyceride (TG) reduction at 6mos., sustained to 12mos., while 89% (50mg) & 88%…

ByRidhi RastogiNovember 10, 2025

NEWS

Johnson & Johnson Reports the US FDA’s Approval of Caplyta (Lumateperone) for Major Depressive Disorder

Shots: The US FDA has approved Caplyta as an adjunctive therapy with antidepressants for treating adults with major depressive disorder (MDD). An sNDA for schizophrenia relapse prevention is also submitted Approval was based on 2 P-III (Study 501 & 502) trials assessing Caplyta vs PBO, both in combination with an antidepressant in MDD pts Trial…

ByRidhi RastogiNovember 7, 2025

NEWS

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

Aqua Medical Receives the US FDA IDE Approval to Initiate Trial for Ablation Procedure to Treat Uncontrolled Type 2 Diabetes

Shots: Aqua Medical has received the US FDA IDE approval to initiate the RESTORE-1 study evaluating the Proximal Intestinal Mucosal Ablation (PIMA) procedure using its radiofrequency vapor ablation (RFVA) system in adults with uncontrolled type 2 diabetes The IDE approval was backed by international data showing safety & metabolic benefits of PIMA, allowing Aqua Medical…

ByRidhi RastogiNovember 6, 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

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

Fresenius Kabi’s Conexxence and Bomyntra (Biosimilars, Prolia & Xgeva) Receives FDA’s Interchangeable Designation

Shots: The US FDA has granted interchangeable designation to Conexxence & Bomyntra, biosimilar versions of Prolia & Xgeva (denosumab), respectively, for the treatment of bone & skeletal disorders Conexxence treats adults at high fracture risk, incl. pts with osteoporosis, pts on long-term glucocorticoid therapy or certain cancer treatments affecting bone density Bomyntra prevents SREs in adults with bone-involved malignancies…

ByRidhi RastogiOctober 30, 2025

NEWS

Avvio Medical’s Avvio Enhanced Lithotripsy System (ELS) Receives the US FDA’s Breakthrough Device Designation (BDD) for Kidney Stone Treatment

Shots: The US FDA has granted BDD to Avvio ELS for minimally invasive, anesthesia-independent kidney stone treatment AVVIO ELS will be investigated in a pivotal ELS trial across the US following IDE approval, with US commercial launch planned in 2026, pending FDA clearance The AVVIO ELS uses microbubble-enhanced acoustic cavitation lithotripsy to enable minimally invasive kidney…

ByRidhi RastogiOctober 29, 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

Ionis Highlights P-III (CORE & CORE2) Trials Results of Olezarsen for Severe Hypertriglyceridemia (sHTG) at AHA 2025

Johnson & Johnson Reports the US FDA’s Approval of Caplyta (Lumateperone) for Major Depressive Disorder

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

Aqua Medical Receives the US FDA IDE Approval to Initiate Trial for Ablation Procedure to Treat Uncontrolled Type 2 Diabetes

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)

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

Fresenius Kabi’s Conexxence and Bomyntra (Biosimilars, Prolia & Xgeva) Receives FDA’s Interchangeable Designation

Avvio Medical’s Avvio Enhanced Lithotripsy System (ELS) Receives the US FDA’s Breakthrough Device Designation (BDD) for Kidney Stone Treatment

New Drug Designations – September 2025

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