Top Regulatory News Stories – Week Ending June 27, 2019

AMAG Pharmaceuticals announced that the U.S. FDA has approved Vyleesi™ (bremelanotide injection), a melanocortin receptor agonist, to treat acquired, generalized hypoactive sexual desire disorder (HSDD) in premenopausal women. https://www.amagpharma.com/news/amag-pharmaceuticals-announces-fda-approval-of-vyleesi-bremelanotide-injection-for-acquired-generalized-hypoactive-sexual-desire-disorder-hsdd-in-premenopausal-women/ Alexion’s drug Ultomiris has snagged a priority review from the FDA to treat atypical hemolytic uremic syndrome (aHUS), an ultra-rare disease that can cause progressive damage[…]

Top Regulatory News Stories – Week Ending June 21, 2019

Blueprint Medicines announced it has submitted a NDA to the FDA for avapritinib for the treatment of adult patients with PDGFRA Exon 18 mutant gastrointestinal stromal tumors (GIST), regardless of prior therapy, and fourth-line GIST.  https://www.drugs.com/nda/avapritinib_190614.html?utm_source=ddc&utm The FDA announced that generic drug applicants will now have access to more information from the agency when deciding[…]

Top Regulatory News Stories – Week Ending June 14, 2019

Genentech’s blood cancer drug Polivy earned an accelerated approval from the U.S. FDA due to the complete response rate shown during clinical trials.  https://www.biospace.com/article/genentech-s-blood-cancer-drug-approved-two-months-ahead-of-pdufa-date/?s=61 The FDA approved Merk’s Keytruda as a frontline treatment for head and neck cancer.  Keytruda was initially approved in 2014 for advanced melanoma and has since received approvals as treatment for[…]

Top Regulatory News Stories – Week Ending June 7, 2019

bluebird bio announced that the European Commission (EC) has granted conditional marketing authorization for ZYNTEGLO™ (autologous CD34+ cells encoding βA-T87Q-globin gene), a gene therapy for patients 12 years and older with transfusion-dependent β-thalassemia (TDT) who do not have a β0/β0 genotype.  https://finance.yahoo.com/news/bluebird-bio-announces-eu-conditional-132500558.html Ridgeback Biotherapeutics LP announced that the FDA has recently granted Orphan Drug Designation[…]

Top Regulatory News Stories – Week Ending May 31, 2019

AveXis/Novartis received FDA approval for Zolgensma, the gene therapy for children younger than two years old who have been diagnosed with SMA with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. The therapy will be available to patients for a cost of $2.125 million, or $425,000 per year for 5 years.  https://www.biospace.com/article/novartis-sma-gene-therapy-treatment-approved-by-the-fda-will-have-a-price-of-2-125-million/ Novocure[…]

Top Regulatory News Stories – Week Ending May 17, 2019

The FDA will be launching an expanded access pilot dubbed ‘Project Facilitate’ by End of May.  This program is aimed at improving patient access to unapproved oncology drugs: https://www.raps.org/news-and-articles/news-articles/2019/5/fda-to-launch-expanded-access-pilot-project-facil?feed=Regulatory-Focus The FDA has granted Breakthrough Therapy Designation for Sunovion’s schizophrenia drug: http://www.wbjournal.com/article/20190513/NEWS01/190519988 Celgene reported that the FDA has granted Breakthrough Therapy designation to POMALYST® (pomalidomide) for[…]

Top Regulatory News Stories – Week Ending May 10, 2019

The FDA approved two oral formulations of the Pfizer drug tafamidis (to be sold as Vyndaqel and Vyndamax) for the treatment of transthyretin amyloidosis (ATTR), a debilitating rare genetic disease. https://xconomy.com/new-york/2019/05/06/fda-oks-pfizer-rare-disease-drug-sets-up-battle-with-alnylam-akcea/?mc_cid=2ebda5e74a&mc_eid=3e3fa57749 Jacobus Pharmaceutical Co won FDA approval for the first drug to treat children with Lambert-Eaton myasthenic syndrome, a rare autoimmune disorder. https://www.reuters.com/article/us-jacobus-fda/fda-grants-jacobus-pharma-approval-for-rare-disease-drug-idUSKCN1SC26U The European[…]

Top Regulatory News Stories – Week Ending May 3, 2019

The U.S. FDA gave Sanofi’s dengue vaccine Dengvaxia a very narrow approval on Wedneday.  The company continues to suffer from evidence that its vaccine, which took 20 years to develop, can cause severe infections in some people.  https://www.reuters.com/article/us-usa-fda-sanofi-fr/sanofi-wins-fda-approval-to-sell-dengue-shot-in-parts-of-u-s-idUSKCN1S74TI?feedType=RSS&feedName=healthNews Finch Therapeutics Group announced that the FDA has granted Fast Track designation to its Full-Spectrum Microbiota® (FSM®)[…]

Top Regulatory News Stories – Week Ending April 26, 2019

Hi folks!  I haven’t done my regulatory news blog in a few months but I’m hoping to make it a regular thing again.  Here are the top regulatory news stories this week: On Tuesday the FDA had approved Abbvie’s Skyrizi (risankizumab-rzaa) for severe plaque psoriasis in adults who are candidates for systemic therapy or phototherapy.[…]

Top Regulatory News Stories – Week Ending December 14, 2018

Novartis’ Sandoz unit and Pear Therapeutics announced Monday that the FDA cleared their prescription-only mobile medical app, reSET-O, marking the first FDA-cleared prescription digital therapeutic for patients with opioid use disorder.  https://www.firstwordpharma.com/node/1610616 Stealth BioTherapeutics announced that the FDA has granted Fast Track designation for its lead investigational candidate, elamipretide, for the treatment of dry age-related[…]