RDR Alert®

Boehringer Ingelheim announced the approval of a new indication for afatinib, as the U.S. FDA approved a supplemental NDA for the first-line treatment of patients with metastatic NSCLC whose tumors have non-resistant EGFR mutations.

HCell announced today that HC017AA, a treatment for Alopecia Areata in pediatric patients, has been granted Orphan Drug Designation by the U.S. FDA.

Filmmaker Kumail Nanjiani opened up on Twitter – perhaps more candidly than ever before – after his wife's mother found an old hospital badge.

Almost exactly one year after it received Orphan Drug Designation in the EU, Benitec has been granted Orphan Drug Designation for its product BB-301, which is being developed to treat oculopharyngeal muscular dystrophy, by the U.S. FDA.

The U.S. Food and Drug Administration has approved a third indication for olaparib tablets (Lynparza).

Results from a study report that behavioral interventions, like environmental enrichment, can reduce the functional deficit experienced by patients with Rett syndrome.

In this Rare Connections program, Martha Raymond; Founder of the Raymond Foundation, Executive Director of Michael’s Mission, and the Co-Chair of the GI Cancers Alliance meets with Sarah Bennett, a cholangiocarcinoma survivor to highlight the space.

Retrophin has committed $3 million over the next 6 years to support the work of the Children’s National Rare Disease Institute, the first Center of Excellence for the National Organization for Rare Disorders.

Roaming monkeys in Florida are being removed by the state’s wildlife managers because of the growing fear that they are excreting a virus that can be dangerous to humans.

At the 36th Annual J.P. Morgan Healthcare Conference, uniQure announced that in 2018, the company intends to advance its gene therapy AMT-061 into a pivotal study for hemophilia B.

After discovering the fate of a former teammate’s child, wrestlers at Wells High School in Maine are using their sport as an incentive to raise money for Sanfilippo Syndrome.

On Monday, Shire announced that it plans to reorganize its business structure and create two distinct operational divisions; one in rare disease and another in neuroscience.

Data published in the Orphanet Journal of Rare Diseases suggests that endurance training could have a positive impact in Huntington’s disease patients, much like it has in anyone else.

Earlier this week, NuMedii, Inc. announced the official formation of an Idiopathic Pulmonary Fibrosis (IPF) Advisory Board.

Avadel Pharmaceuticals announced this morning that FT 218, its once-nightly product for the treatment of narcolepsy, has been granted Orphan Drug Designation by the U.S. Food and Drug Administration.

On Tuesday, University of Central Florida (UCF) senior linebacker Shaquem Griffin became the 10th winner of the prestigious Rare Disease Champion Award.

BioMarin Chief Executive Officer Jean-Jacques Bienaime spoke about the company’s development of valoctocogene roxaparvovec, a gene therapy in development for the treatment of Hemophilia A.

Global Blood Therapeutics, Inc. was granted Breakthrough Therapy Designation from the U.S. Food and Drug Administration for voxelotor for the treatment of sickle cell disease.

Adamas Pharmaceuticals, Inc. announced the full commercial launch of Gocovri (amantadine) extended release capsules for the treatment of dyskinesia in patients with Parkinson’s disease receiving levodopa-based therapy, with or without concomitant dopaminergic medications.

Acceleron Pharma has announced that Part 1 of the Phase 2 clinical trial of ACE-083, a drug used to increase muscle volume in facioscapulohumeral dystrophy (FSHD), has exhibited positive results.

Stephen Hawking, the notable British physicist and author of A Brief History of Time, celebrates his 76th birthday today, making him one of the longest surviving patients with amyotrophic lateral sclerosis (ALS).

Last week, Pfizer announced a new partnership with Sangamo. As part of the agreement, the sides will team to develop a potential gene therapy to treat ALS, or Lou Gehrig’s disease.

Two months after Ionis submitted its NDA to the U.S. FDA, the company has announced that the regulatory agency has accepted its investigational drug inotersen for Priority Review.

This morning, it was announced that Retrophin, Inc. and Censa Pharmaceuticals are partnering to continue development and evaluate CNSA-001 for the treatment of phenylketonuria (PKU).

Mallinckrodt plc has completed the rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA), as per an official announcement from the company yesterday.

Last night, the U.S. FDA posted a warning letter addressed to American CryoStem Corporation for marketing an adipose derived stem cell product without FDA approval and for significant deviations from current good manufacturing practice requirements.

NORD announced that it will commemorate the 35th anniversaries of both its formation and the creation of the Orphan Drug Act with a year-long commemoration.

Catalyst Biosciences announced the initiation and open enrollment of the Phase 2 part of its Phase 2/3 program of marzeptacog alfa (activated), a highly potent, subcutaneously administered Factor VIIa therapy in development for the treatment of hemophilia A and B with inhibitors.

This morning, the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation to Novatris’ eltrombopag (Promacta), a first-line treatment for severe aplastic anemia (SAA).

Recent data have found that the same gene editing platform can disable the defective gene responsible for amyotrophic lateral sclerosis in mice. The therapy, which extended the lifespan of the mouse models by 25%, delayed the onset of the muscle wasting which characterizes the disease.