Home / Rare Diseases (page 20)

Rare Diseases

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 …

Read More »

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 …

Read More »

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 …

Read More »

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BOULDER, Colo., March 19, 2017 /PRNewswire/ — Array BioPharma Inc. (Nasdaq: ARRY) today announced that it has withdrawn from the U.S. Food and Drug Administration’s (FDA) Division of Oncology Products 2 its new drug application (NDA) for binimetinib monotherapy for the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

Read More »

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 …

Read More »

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 …

Read More »

Allergan and Editas Announce CRISPR-Based Research and Development Deal for Rare Eye Diseases

DUBLIN and CAMBRIDGE, Mass., March 14, 2017 /PRNewswire/ — Allergan plc (NYSE: AGN), a leading global pharmaceutical company, and Editas Medicine, Inc. (NASDAQ: EDIT), a leading genome editing company, today announced that Allergan’s wholly-owned subsidiary, Allergan Pharmaceuticals International Limited, and Editas Medicine have entered into a strategic research and development alliance …

Read More »

FDA Approves Increased Shelf Life and In-Use Storage at Room Temperature for Orfadin

WALTHAM, Mass.–(BUSINESS WIRE)–Sobi, a leading integrated biopharmaceutical company dedicated to bringing innovative therapies and services to improve the lives of rare disease patients and their families, announced today that the FDA approved a supplemental new drug application (sNDA) for Orfadin® (nitisinone) capsules in the U.S. The sNDA will allow for …

Read More »

Exonics Therapeutics Launches with Funding from CureDuchenne to Advance CRISPR/Cas9 Therapy for DMD

BOSTON, Feb. 27, 2017 (GLOBE NEWSWIRE) — Exonics Therapeutics, Inc., a newly formed biotechnology company focused on developing gene editing technologies like CRISPR/Cas9 to permanently correct a majority of mutations causing Duchenne muscular dystrophy and other neuromuscular diseases, today announced a commitment of $5 million in seed financing from CureDuchenne …

Read More »

Sangamo Therapeutics Receives Rare Pediatric Disease Designation from the FDA for its MPS I Candidate

RICHMOND, Calif., Feb. 27, 2017 /PRNewswire/ — Sangamo Therapeutics, Inc. (Nasdaq: SGMO), the leader in therapeutic genome editing, today announced that the U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation for SB-318, the Company’s in vivo genome editing product candidate for the treatment of Mucopolysaccharidosis Type …

Read More »