Rare Diseases Archives - Page 15 of 15

FDA Grants Breakthrough Therapy Designation for Tafamidis for the Treatment of Patients with Transthyretin Cardiomyopathy

Breakthrough Therapy Status, FDA, Progressive Heart Failure, Transthyretin Cardiomyopathy

Pfizer Inc. announced that tafamidis received Breakthrough Therapy designation from the U.S. Food and Drug Administration for the treatment of patients with transthyretin cardiomyopathy, which is a rare, fatal and underdiagnosed condition associated with progressive heart failure.

May 23, 2018/by Business Wire

FDA Grants Priority Review for Lanadelumab

Biologics License Application (BLA), FDA/Regulatory, Hereditary Angioedema (HAE), Monoclonal Antibodies, Plasma Kallikrein (pKal) Inhibitors, Priority Review, Rare Diseases

Shire plc announced the U.S. Food and Drug Administration accepted the Biologics License Application and granted priority review for lanadelumab (SHP643).

February 23, 2018/by Administrator

Shire, Microsoft and EURORDIS form Global Commission to accelerate time to diagnosis for children with rare diseases

Alliances, Children, Healthcare Providers, Innovation, Patient Advocates, Rare Diseases, Researchers, Technology

Shire, Microsoft and EURORDIS-Rare Diseases Europe announced a strategic alliance to address the diagnostic challenge for patients living with a rare disease.

February 20, 2018/by Administrator

Alnylam and Sanofi Enter into Strategic Restructuring of RNAi Therapeutics Rare Disease Alliance

Alliances, Biology, Business, Drug Development, Gene Silencing, Genetic Diseases, Hereditary ATTR Amyloidosis, Product Pipelines, RNA Interference (RNAi)

Alnylam and Sanofi announced a strategic restructuring of their RNAi therapeutics alliance to streamline and optimize development and commercialization of certain products for rare genetic diseases.

January 7, 2018/by Business Wire

UDG Healthcare announces acquisition of Cambridge BioMarketing for up to $35M

Business, Marketing & Advertising, Orphan Drugs, Rare Diseases

UDG Healthcare plc announced the acquisition of Cambridge BioMarketing LLC, a U.S.-based healthcare communications business, for a total consideration of up to $35 million.

July 17, 2017/by Administrator

EU Approves Brineura for CLN2 Disease

Approvals, EMA, European Commission, FDA/Regulatory, First-In-Class, Neuronal Ceroid Lipofuscinosis Type 2 (CLN2), Rare Diseases

The European Commission granted marketing authorization for BioMarin Pharmaceutical’s Brineura for the treatment of tripeptidyl peptidase 1 (TPP1) deficiency.

June 1, 2017/by Andrew Humphreys

Incentives, Opportunities Abound as Orphan Drug Demand Grows

Biopharma, Bones, Breakthrough Therapy Designation, CDC, Congress, Department of Health and Human Services (HHS), EMA, Enzyme Replacement, EU, FDA, FDA/Regulatory, glioblastoma Multiforme (GBM) Brain Cancer, Hypophosphatasia (HPP), June 2017, Metabolic Disorders, Orphan Drug Act, Rare Diseases

Concerted effort by governments, regulators, and industry is rapidly expanding development of new therapies for the more than 7,000 known rare diseases.

June 8, 2016/by Administrator

New Jersey Rare Disease Alliance Commemorates Global Rare Disease Day

Health, Rare Diseases

Hosts February 29 Event on Building Relationships and Raising Awareness Ramsey, NJ (February 19, 2016) The New Jersey Rare Disease Alliance – dedicated to improving the lives of the […]

February 24, 2016/by Administrator

More than half of the world’s population will live in countries where medicine use will exceed one dose per person per day by 2020, up from 31 percent in 2005, as the “medicine use gap” between developed and pharmerging markets narrows, according to research by the IMS Institute for Healthcare Informatics.

December 10, 2015/by Joshua Slatko