RDR Alert®

Arrowhead Pharmaceuticals Inc. announced that the company’s second-generation investigational medicine ARO-AAT was granted orphan drug designation by the FDA.

Celgene Corporation announced that data from the Phase 3 RELIEF clinical trial of Otezla (apremilast) in patients with active Behçet’s disease with oral ulcers were presented.

Multiple Phase 3 studies that evaluated the treatment of Dysport in sustained spasticity patients will be presented at the annual meeting of the Association of Academic Physiatrists.

Akcea announced that the Division of Metabolism and Endocrinology Products of the U.S. FDA will hold an advisory committee meeting to review data supporting the NDA for volanesorsen.

AstraZeneca and Merck announced that selumetinib, its MEK 1/2 inhibitor for the treatment of NF1, was granted orphan drug designation by the U.S. FDA.

Patricia Dickson, M.D., discusses similarities between variations of MPS and the both the benefits and challenges of being a researcher in the rare disease community.

Late last night, U.S. FDA Commissioner Scott Gottlieb released a statement regarding the Administration’s request for new FDA funding to “promote innovation and broaden patient access through competition.”

A researcher at the Feinstein Institute for Medical Research has been awarded two grants for clinical trials on existing FDA-approved drugs that can be indicated to treat hereditary hemorrhagic telangiectasia.

Genentech announced that its Supplemental Biologics License Application was accepted, and the FDA granted Priority Review for the use of rituximab for the treatment of pemphigus vulgaris.

On February 27, Marquette University’s College of Health Sciences will host a discussion led by Harmony 4 Hope and other rare disease advocates in honor of rare disease day.

Data published in The Orphanet Journal of Rare Diseases confirmed that currently-available enzyme replacement therapies are unable to reverse or stabilize the progression of Pompe disease.

Alexy V. Pshezhetsky, Ph.D., professor at the University of Montreal. sits down at WORLDSymposium to stress the importance of researcher involvement throughout the rare disease community.

Imara has announced that it has dosed the first patient in its Phase 2a clinical trial of IMR-687 in adult patients with sickle cell disease.

Catabasis Pharmaceuticals reported positive safety and efficacy results, displaying the preservation of muscle function and sustained disease-modifying effects in Duchenne muscular dystrophy with edasalonexent.

Hartmann Wellhoefer, M.D., head of medical affairs at Shire, sits down with RDR at the 14th Annual WORLDSymposium to discuss the company's recent data for lysosomal storage diseases.

The U.S. FDA approved tezacaftor/ivacaftor and ivacaftor (Symdeko) for the treatment of the underlying cause of cystic fibrosis in people ages 12 years and older who have 2 copies of the F508del mutation.

Audentes Therapeutics has initiated the first patient dosing of VALENS, a Phase 1/2 clinical trial of a potential treatment for Crigler-Najjar syndrome called AT342.

Xeris announced this morning that the U.S. FDA has granted Orphan Drug designation to the ready-to-use, liquid-stable glucagon for the treatment of Hyperinsulinemic Hypoglycemia

Teva has announced the release of a generic version of Syprine (trientine hydrochloride) capsules, 250 mg, as a substitute treatment for patients with Wilson’s disease in the United States.

The International Foundation for Functional Gastrointestinal Disorders will participate in Rare Disease Day on February 28, with efforts to support patients affected by a rare digestive disease globally.

Abeona Therapeutics, Inc. announced that the FDA has granted Orphan Drug Designation to its ABO-202 program for the treatment of infantile Batten disease.

Amicus announced that the U.S. FDA has accepted its New Drug Application submitting for filing under priority review for migalastat for the treatment of patients with Fabry disease with amenable mutations.

With no real standard-of-care for Sanfilippo syndrome patients, Brian Bigger, Ph.D., discusses the sense of urgency he and his colleagues feel while researching.

BioMarin presented interim data at the 14th Annual WORLDSymposium in San Diego from a Phase 1/2 trial for BMN 250 in MPS IIIB.

ArmaGen reported full 52-week results from a Phase 2 proof-of-concept study with AGT-181 in MPS I.