Hypophosphatasia (HPP)

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.

Alexion’s $550,000 bone drug too expensive for Britain

Health, Hypophosphatasia (HPP), Metabolic Bone Disorders, Metabolic Disorders, National Institute for Health and Care Excellence (NICE), Pricing, Rare Diseases, Therapeutics, Watchdog

A new drug to treat a rare inherited bone disorder from Alexion Pharmaceuticals ALXN.O that costs more than $550,000 a year for each patient has been deemed too expensive by […]

FDA approves Alexion’s metabolic bone disorder drug

Approvals, FDA/Regulatory, First-In-Class, Health, Hypophosphatasia (HPP), Japan, Metabolic, Metabolic Bone Disorders, Metabolic Disorders, New Drug Approvals, Shares, Shares, Skeletal, Skeletal Abnormalities

Oct 23 (Reuters) – The U.S. Food and Drug Administration on Friday approved Alexion Pharmaceuticals Inc’s Strensiq, a treatment for a rare kind of metabolic bone disorder. The approval is […]