Pfizer’s oral Janus kinase 1 (JAK1) inhibitor abrocitinib hit the primary endpoint in a second Phase III study in patients 12 and older with moderate to severe atopic dermatitis.
Shares of Acadia Pharmaceuticals were up nearly 70 percent after a Phase III psychosis trial was halted early as the Parkinson’s disease drug Nuplazid hit primary endpoints, positioning the company to seek another potential regulatory approval.
BioMarin’s Hemophilia A Gene Therapy Shows Promise in Clinical Trial
Analysts, Breakthrough Therapy Status, Clinical Data, Clinical Trials, European Medicines Agency, European Medicines Agency (EMA), Factor VIII Inhibitors, FDA, Gene Therapy, Hemophilia A, Orphan Drug Designation, Priority Medicines (PRIME) access, R&DSan Rafael, Calif.-based BioMarin Pharmaceutical announced positive Phase III clinical trial results for the company’s gene therapy, valoctocogene roxaparvovec, for adults with hemophilia A.
Pfizer Inc.’s experimental atopic dermatitis treatment abrocitinib met the main goals in a late-stage study that tested the drug in patients aged 12 and older with moderate to severe forms of the disease.
AstraZeneca’s Calquence (acalabrutinib) hit the mark in a Phase III trial of previously treated patients with chronic lymphocytic leukemia (CLL).
The obscure advisory committees at the heart of the U.S. drug pricing debate
Breakthrough Therapy Status, Congress, CVS Health, Diabetes, Donald Trump, Drug Prices, Duchenne Muscular Dystrophy (DMD), Express Scripts Holding, FDA, Migraine, Multiple Sclerosis, Pharmacy Benefit Managers, Spinal Muscular Atrophy (SMA), UnitedHealth GroupExpectations were high during 2018 for three new migraine drugs hitting the market from Amgen Inc., Eli Lilly and Co. and Teva Pharmaceutical Industries Ltd. Priced around $7,000 each, the drugmakers called them “breakthrough” treatments designed to prevent migraines when taken year-round, and estimated that millions of patients could benefit. But a small group of medical experts who quietly advise U.S. health insurers on new drugs was not impressed, according to a private meeting held at UnitedHealth Group’s OptumRx offices in Chicago that was attended by Reuters.
Horizon Pharma announced that new data from the Phase 3 confirmatory study evaluating teprotumumab for treating active thyroid eye disease were presented as part of a late-breaking oral presentation at the 2019 AACE Scientific and Clinical Congress.
Teva Pharmaceutical Industries Ltd. will stop developing the migraine drug Ajovy for treating cluster headaches after finding the treatment was unlikely to meet the main goal of a late-stage trial.
Galera Therapeutics Inc.announced that the clinical-stage biotechnology company secured $150 million in a joint, oversubscribed Series C financing and royalty purchase agreement.
Pfizer rare heart disease drug reduces risk of death by 30 percent in study
Breakthrough Therapy Status, Clinical Data, Clinical Studies, Clinical Trials, Familial Amyloid Polyneuropathy, FDA, Heart Disease, Hereditary TTR Amyloidosis (hATTR), Neurodegenerative Diseases, Potential Blockbusters, R&D, Transthyretin Amyloid Cardiomyopathy (ATTR-CM)Pfizer Inc.’s tafamidis reduced the risk of death for patients with a rare and fatal heart disease by around 30 percent, boosting the prospects of what could be a billion-dollar-a-year drug.
