Tag: medicinal product

BEERSE, BELGIUM, June 26, 2026 (GLOBE NEWSWIRE) -- Johnson & Johnson today announced new data across 12 abstracts at the European Academy of Neurology (EAN) 2026 Congress that offer additional insight into the use of IMAAVY^®▼(nipocalimab) throughout clinically relevant points in the generalised myasthenia gravis (gMG) treatment journey. The analyses include adults with anti-AChR^a or anti-MuSK^b antibody-positive gMG who were early in their disease course or had lower baseline symptom burden – providing insight into the potential importance of addressing pathogenic immunoglobulin G (IgG) early in disease progression where use of advanced therapies may be less common.^1,2 Additional research to be shared include outcomes shortly after common infections, which are a known cause of disease exacerbations in gMG, and plans to address evidence gaps in the use of nipocalimab during pregnancy.^3,4

SAN DIEGO, June 26, 2026 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for rare diseases, today announced positive five-year data from its ongoing HOPE-2 Open-Label Extension (OLE) study of Deramiocel, the Company’s lead cell therapy candidate for Duchenne Muscular Dystrophy (DMD). The data will be presented at the Parent Project Muscular Dystrophy (PPMD) 2026 Annual Conference, taking place June 25-27, 2026, in Orlando, Florida, alongside previously reported results from the Company’s HOPE-3 Phase 3 trial.

FLORENCE, Italy and NEW YORK, June 14, 2026 (GLOBE NEWSWIRE) -- The Menarini Group (“Menarini”), a leading international pharmaceutical and diagnostics company, and Stemline Therapeutics, Inc. (“Stemline”), a wholly-owned subsidiary of the Menarini Group focused on bringing transformational oncology treatments to cancer patients, announced that new data related to the pivotal Phase 3 SENTRY trial will be presented as a late-breaking oral at The European Hematology Association (EHA) 2026 Congress.

Warsaw, Poland – June 03, 2026 – JJP Biologics, ("JJPBio" or the "Company") a clinical-stage, immune-focused biotech that engineers precision antibodies to correct derailed immune pathways that drive autoimmune diseases and cancer, today announces positive interim data from its ongoing Phase 1b trial evaluating nebaprubart, also known as JJP-1212, its investigational potential first-in-class anti-CD89 antagonist, in patients diagnosed with Linear IgA Disease (LAD), a rare autoantibody-mediated skin disease.

Pratteln, Switzerland, 13 August, 2025 – Santhera Pharmaceuticals (SIX: SANN) announces the signing of an exclusive agreement with Gen İlaç ve Sağlık Ürünleri San. ve Tic. A.Ş. (GEN) for the distribution and promotion of AGAMREE® (vamorolone) in Türkiye for the treatment of Duchenne muscular dystrophy (DMD) in patients four years of age and older.

CELEBRATION, Fla., July 28, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare diseases, announced the company submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for the evaluation of arimoclomol for the treatment of Niemann-Pick Disease Type C (NPC). NPC is an ultra-rare, neurological disease caused by genetic mutations that result in lipid accumulation in cells, leading to visceral, neurological, and psychiatric symptoms. Arimoclomol is the only treatment shown to directly target the underlying pathology of NPC by increasing gene expression for improved lipid clearance. Arimoclomol for the treatment of NPC has been designated as an Orphan Medicinal Product by the EMA. Arimoclomol is marketed in the U.S. under the brand name MIPLYFFA®.

Complete Response Letter (CRL) cited specific chemistry, manufacturing and controls (CMC) related observations that are resolvable

PARIS and CAMBRIDGE, Mass., July 07, 2025 (GLOBE NEWSWIRE) -- NANOBIOTIX (Euronext: NANO –– NASDAQ: NBTX – the ‘‘Company’’),  a late-stage clinical biotechnology company pioneering physics-based approaches to expand treatment possibilities for patients with cancer, today announced two important developments that aim to reinforce the global positioning of potential first-in-class radioenhancer JNJ-1900 (NBTXR3), which is licensed by Janssen Pharmaceutica NV, a Johnson & Johnson company.