Leadiant Biosciences that the FDA has granted approval to Revcovi (elapegademase-lvlr) injection in the U.S. Revcovi is a new enzyme replacement therapy (ERT) for the treatment of adenosine deaminase severe combined immune deficiency (ADA-SCID) in pediatric and adult patients. “We are gratified by the FDA’s timely recognition of Revcovi as an effective and safe treatment for ADA-SCID,…
FDA Approves New Drug for Treatment of the Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosi
the US Food and Drug Administration (FDA) approved Tegsedi (inotersen), an antisense oligonucleotide (ASO) that inhibits the production of the transthyretin (TTR) protein (amyloid), for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR) in adults. This is the second FDA approved drug for hATTR. Onpattro (patisiran), was the first treatment approved for this indication…
Orchard Therapeutics Receives EMA PRIME Designation for Investigational Lentiviral Gene Therapy
Orchard Therapeutics, a leading commercial-stage biotech company dedicated to transforming the lives of patients with rare diseases through innovative gene therapies, today announced that the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to OTL-300, an investigational autologous ex vivo lentiviral gene therapy for the treatment of transfusion-dependent beta-thalassemia (TDBT), the most severe…
FDA Approves Antibacterial Drug to Treat Serious Lung Disease Using a Novel Pathway
The FDA approved a new drug, Arikayce (amikacin liposome inhalation suspension), for the treatment of lung disease caused by a group of bacteria, Mycobacterium avium complex (MAC) in a limited population of patients with the disease who do not respond to conventional treatment (refractory disease). MAC is a type of nontuberculous mycobacteria (NTM) commonly found…
FDA Approves Antibacterial Drug for Serious Lung Disease
FDA Awards 12 Grants for Drug Development for Rare Diseases
European Agency Recommends Novartis’ Gene Therapy for Rare Inherited Disorder
The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended granting a marketing authorization for the gene therapy Luxturna (voretigene neparvovec), for the treatment of adults and children suffering from inherited retinal dystrophy caused by RPE65 gene mutations, a rare genetic disorder which causes vision loss and usually leads to blindness. The mutations of…
FDA Approves New Kind of Treatment for Hairy Cell Leukemia
Yeast Research Leads to Central Nervous System Disorder Finding
FDA Approves AstraZeneca’s Treatment for Hairy Cell Leukemia
The FDA approved Lumoxiti (moxetumomab pasudotox-tdfk) injection for intravenous use for the treatment of adult patients with relapsed or refractory hairy cell leukemia (HCL) who have received at least two prior systemic therapies, including treatment with a purine nucleoside analog. Lumoxiti is a CD22-directed cytotoxin and is the first of this type of treatment for…
How a Collaborative Ecosystem is Driving the Development of Successful Therapies in Rare Disease Research
In total, approximately 7,000 rare diseases have been identified—with many more being discovered every year. While a single orphan disease may affect just a handful of individuals, the global impact is significant: in the United States alone between 25 and 35 million people are estimated to be diagnosed with a rare disease, with 50-66 percent…
EU Regulators Approve Sanofi’s Cablivi
The European Commission has granted marketing authorization for Cablivi (caplacizumab) for the treatment of adults experiencing an episode of acquired thrombotic thrombocytopenic purpura (aTTP), a rare blood-clotting disorder. Cablivi is the first therapeutic specifically indicated for the treatment of aTTP. aTTP is a life-threatening, autoimmune-based blood clotting disorder characterized by extensive clot formation in small blood…
Blood Pressure Drug Slows Aortic Dilatation in Marfan Syndrome
Microvascular Dysfunction: A Common Cause of Heart Failure
Researchers Discover First Treatment to Improve Survival in Rare Heart Condition
Pfizer’s Tafamidis Phase 3 Study Shows Reduced Risk of Transthyretin Cardiomyopathy Mortality
The primary results from Pfizer’s Tafamidis Phase 3 Transthyretin Cardiomyopathy (ATTR-ACT) study, which showed tafamidis significantly reduced the hierarchical combination of both all-cause mortality and frequency of cardiovascular-related hospitalizations compared to placebo over a 30-month period (P=0.0006) in patients with wild-type or variant (hereditary) transthyretin amyloid cardiomyopathy (ATTR-CM).1 ATTR-CM is a rare, fatal, and underdiagnosed condition…
Akcea and Ionis Receive FDA Rejection for Waylivra
Akcea Therapeutics, Inc. an affiliate of Ionis Pharmaceuticals, Inc., and Ionis Pharmaceuticals, Inc., received a Complete Response Letter (CRL) from the Division of Metabolism and Endocrinology Products of the U.S. Food and Drug Administration (FDA) regarding the New Drug Application (NDA) for Waylivra (volanesorsen). “We are extremely disappointed with the FDA’s decision. FCS is an ultra-rare and debilitating…
Scientists Closer to Solving Arthritic Condition in Teens
Centogene and Evotec Sign Global Strategic Partnership on Drug Discovery Projects
Evotec Centogene have entered into a global strategic collaboration agreement for joint drug discovery projects, developing compounds to treat rare genetic diseases. Centogene and Evotec initiated the collaboration to develop a strategic high-throughput platform for testing novel small molecules in rare hereditary metabolic diseases. The collaboration brings together Evotec’s leading induced pluripotent stem cell (“iPSC”) platform…
Diacomit Approved for Treatment of Dravet Syndrome Seizures
The FDA approved Diacomit (stiripentol) for the treatment of seizures associated with Dravet syndrome, a rare form of epilepsy. Diacomit is indicated for use in patients two years of age and older taking clobazam. There are no clinical data to support the use of Diacomit as monotherapy in Dravet syndrome. Dravet syndrome is a rare genetic…
FDA Accepts Priority Review of ALXN1210 for Treatment of Rare Blood Disorder
Alexion Pharmaceuticals, Inc. announced that the FDA has accepted for review the Company’s Biologics License Application (BLA) for approval of ALXN1210, the Company’s investigational long-acting C5 complement inhibitor, for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH). The FDA set a Prescription Drug User Fee Act (PDUFA) date of February 18, 2019, as part…
Statins Associated With Improvement of Rare Lung Disease
FINDINGS In the first study of its kind, researchers have found that cholesterol-lowering statins may improve the conditions of people with a rare lung disease called autoimmune pulmonary alveolar proteinosis. The research also suggested that two new tests could help diagnose the condition. BACKGROUND Autoimmune pulmonary alveolar proteinosis is a rare syndrome affecting 1 in…
Martin Pharmaceuticals’ Linvantra Receives FDA Orphan Drug Designation in Second Indication
Martin Pharmaceuticals, a clinical stage pharmaceutical company focused on repurposing already-approved drugs to offer life-changing advances to patients afflicted with rare (orphan) diseases or challenging medical conditions, has been granted Orphan Drug Designation by the FDA for Livantra in the treatment of Pulmonary Arterial Hypertension. Earlier this year Livantra received orphan drug designation for the treatment…
FDA Approves New Treatment For Rare Genetic Disorder
Amicus Therapeutics has received approval for Galafold (migalastat), the first oral medication for the treatment of adults with Fabry disease. The drug is indicated for adults with Fabry disease who have a genetic mutation determined to be responsive (“amenable”) to treatment with Galafold based on laboratory data. Fabry disease is a rare and serious genetic…
FDA Approves Onpattro, First-Of-Its Kind Targeted RNA-Based Therapy
The FDA approved Onpattro (patisiran) infusion for the treatment of peripheral nerve disease (polyneuropathy) caused by hereditary transthyretin-mediated amyloidosis (hATTR) in adult patients. This is the first FDA-approved treatment for patients with polyneuropathy caused by hATTR, a rare, debilitating and often fatal genetic disease characterized by the buildup of abnormal amyloid protein in peripheral nerves,…