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Rare Diseases

Akashi Announces FDA Gives Okay to Resume Clinical Development of its DMD Drug

CAMBRIDGE, Mass., March 22, 2017 – Akashi Therapeutics, Inc. a clinical stage biopharmaceutical company developing treatments for patients with Duchenne muscular dystrophy (DMD), today reported that the U.S. Food and Drug Administration (FDA) has completed its review and concluded that Akashi may resume clinical development of HT-100 (delayed-release halofuginone) in …

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Facial Recognition Software Helps Diagnose Rare Genetic Disease

Researchers with the National Human Genome Research Institute (NHGRI), part of the National Institutes of Health, and their collaborators, have successfully used facial recognition software to diagnose a rare, genetic disease in Africans, Asians and Latin Americans. The disease, 22q11.2 deletion syndrome, also known as DiGeorge syndrome and velocardiofacial syndrome, …

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FDA Grants Breakthrough Therapy Designation to Genentech’s Rituxan for Treatment of Pemphigus Vulgaris

SOUTH SAN FRANCISCO, Calif.–(BUSINESS WIRE)–Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation status to Rituxan® (rituximab) for pemphigus vulgaris, a rare, serious and life-threatening condition characterized by progressive painful blistering of …

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FDA Grants Accelerated Approval to Bavencio, the First Treatment for Rare Form of Skin Cancer

The U.S. Food and Drug Administration today granted accelerated approval to Bavencio (avelumab) for the treatment of adults and pediatric patients 12 years and older with metastatic Merkel cell carcinoma (MCC), including those who have not received prior chemotherapy. This is the first FDA-approved treatment for metastatic MCC, a rare, …

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CSL Behring’s CSL830 Prevents Hereditary Angioedema Attacks in Late-Stage Study

Median reduction in hereditary angioedema (HAE) attack rate relative to placebo was 95% at the highest dose studied Data show up to 40% of HAE patients were free from attacks throughout the study In the clinical study, CSL830 met the primary endpoint for preventing HAE attacks Global biotherapeutics leader CSL …

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Shire’s Hereditary Thrombotic Thrombocytopenic Purpura Treatment Receives Fast Track Status from the FDA

Lexington, Mass. – March 22, 2017 – Shire plc (LSE: SHP, NASDAQ: SHPG) today announced that the United States Food and Drug Administration (FDA) has granted Fast Track designation for recombinant ADAMTS13 (SHP655 – historically known as BAX930) for treatment of acute episodes of hereditary thrombotic thrombocytopenic purpura (hTTP) in …

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FDA Grants Orphan Drug Status to NantKwest’s Merkel Cell Carcinoma Candidate

CULVER CITY, Calif.–(BUSINESS WIRE)–NantKwest, Inc. (Nasdaq:NK), a pioneering, next generation, clinical-stage immunotherapy company focused on harnessing the unique power of our immune system using natural killer (NK) cells to treat cancer, infectious diseases and inflammatory diseases, announced today that the FDA has granted Orphan Drug Designation to the company’s activated …

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Array BioPharma Withdraws NDA for its Candidate for Treatment of a Rare Type of Skin Cancer

BOULDER, Colo., March 19, 2017 /PRNewswire/ — Array BioPharma Inc. (Nasdaq: ARRY) today announced that it has withdrawn from the U.S. Food and Drug Administration’s (FDA) Division of Oncology Products 2 its new drug application (NDA) for binimetinib monotherapy for the treatment of NRAS-mutant melanoma, a rare, mutationally-driven subset of skin …

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AveXis’ Gene Therapy for SMA Demonstrates Positive Results

CHICAGO, March 16, 2017 (GLOBE NEWSWIRE) — AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today reported topline results from the Phase 1 trial of AVXS-101 in spinal muscular atrophy (SMA) Type 1. The company also reported financial …

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PTC Therapeutics Acquires Marathon Pharma’s DMD Drug Emflaza for $140 Million

SOUTH PLAINFIELD, N.J., March 16, 2017 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced it has entered into an asset purchase agreement with Marathon Pharmaceuticals, LLC to acquire all rights to Emflaza™ (deflazacort). Emflaza is the first treatment approved in the United States for all Duchenne muscular dystrophy (DMD) patients …

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