RDR Alert®

The FDA has accepted for review supplemental Biologics License Application for Ruconest for routine prophylaxis to prevent attacks in adult and adolescent patients with hereditary angioedema, Pharming Group N.V. reported this morning.

Ipsen and Exelixis, Inc. announced detailed results of the pivotal phase 3 CELESTIAL trial, evaluating cabozatinib in previously treated patients with advanced hepatocellular carcinoma.

NORD, the leading nonprofit organization dedicated to helping Americans with rare diseases, announced this morning that its 7,000 Mile Rare Movement will be kicking off this year’s celebration.

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.

Today, Eiger BioPharmaceuticals announced that results from the Phase 2 LIBERTY study demonstrated no improvement overall in its Phase 2 PAH treatment study, evaluating the effects of ubenimex.

Researchers at St. Jude Children’s Research Hospital have developed a new gene therapy that creates fully-functioning immune systems in babies diagnosed with severe combined immunodeficiency, commonly referred to as the “Bubble Boy” disease.

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.

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.

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.

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.

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.

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).