This morning, Sobi, announced that the FDA has issued a Study may proceed letter for the first study in humans, thereby accepting the investigational new drug application for sobi003, and the regulatory agency granted Fast Track status to the product candidate.
Marshall Summar, M.D., director of the Children’s National Rare Disease Institute, sat down with Rare Disease Report to discuss recent news and what it means for the rare disease community.
To kick off the new year, Alnylam Pharmaceuticals, Inc., a company dedicated to the development of RNAi therapeutics, announced the expansion of its Alnylam Act.
uniQure was granted Orphan Medicinal Product Designation from the European Medicines Agency for AMT-130, an investigational gene therapy for the treatment of Huntington’s disease.
Engineers from the Engineering Faculty at the University of Sheffield and Boston Children’s Hospital, Harvard Medical School have created a robot with the intention of esophageal atresia .
Arimoclomol, in development by Danish biotech Orphazyme A/S for the treatment of Niemann-Pick Disease Type C, was granted rare pediatric disease designation by the U.S. FDA this morning.
New research published in Annals of Internal Medicine shows that the rate of arterial and venous thrombosis was significant in patients with myeloproliferative neoplasms (MPNs).
In 2018, uniQure N.V. expects to advance the clinical development of AMT-130, its investigational gene therapy for the treatment of Huntington’s disease.
Last night, the U.S. FDA approved the supplemental New Drug Application to add overall survival data from the Phase 3 head-to-head ENDEAVOR trial to the Prescribing Information for Kyprolis (carfilzomib).
This morning, Novartis was granted U.S. FDA Priority Review for the drug in patients with relapsed or refractory diffuse large B-cell lymphoma who are ineligible for or relapse after autologous stem cell transplant (ASCT).
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.
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.
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.
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.
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.
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.
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.