Hi folks. This week a landmark gene therapy to treat blindness got one step closer to FDA approval with a resounding panel endorsement. And the FDA issued a disappointing CRL for an acute pain medication. Here are the top regulatory news stories for pharma and biotech this week:
Flexion Therapeutics secured approval from the US FDA for its injectable treatment for osteoarthritis knee pain. The drug, which will be marketed as Zilretta, combines the commonly used corticosteroid triamcinolone acetonide with a drug delivery system designed to provide extended pain relief over three months. http://www.biopharmadive.com/news/flexion-osteoarthritis-FDA-approval-zilretta/506810/
Spark Therapeutics took its gene therapy for blindness “Luxturna” before the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee on Thursday. The panel voted 16 to 0 in favor of approving the subretinal injection, representing a major step forward for adeno-associated virus gene therapies. http://raps.org/Regulatory-Focus/News/2017/10/12/28680/FDA-Panel-Votes-Unanimously-in-Favor-of-Gene-Therapy-to-Treat-Rare-Eye-Disorder/
Prometic Life Sciences announced that the FDA has accepted its BLA for its plasminogen replacement therapy (RyplazimTM). Having priority review status they now have a PDUFA action date for April 14, 2018. Ryplazim™ had previously been granted Fast Track, Orphan Drug and Rare Pediatric Disease designations by the U.S. FDA. http://www.prometic.com/prometic-announces-fda-acceptance-of-its-biologics-license-application-for-plasminogen-ryplazim/
FDA granted Orphan Drug Designation to Trovagene’s PLK1 Inhibitor, PCM-075, for the treatment of Acute Myeloid Leukemia. https://www.firstwordpharma.com/node/1511236
The FDA has rejected AcelRx Pharmaceuticals’ pain medication, Dsuvia. The agency’s complete response letter has specified the need for more safety data and it also suggested changing the administration instructions to make them clearer. http://www.biopharmadive.com/news/acelrx-dsuvia-rejection-fda-crl-pain/507152/
Ardelyx has reported that they plan to submit an NDA in the second half of 2018 for their IBS drug, Tenapanor, which saw positive results in a Phase 3 trial: http://www.biopharmadive.com/news/ardelyx-ibs-drug-edges-closer-to-the-market/507101/
So far in 2017 the FDA has approved 34 new molecular entities through CDER https://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugInnovation/ucm537040.htm and an additional 14 new vaccine/blood/biologics programs through CBER https://www.fda.gov/BiologicsBloodVaccines/DevelopmentApprovalProcess/BiologicalApprovalsbyYear/ucm547553.htm
In other news…
Merck is buying into biotech player KalVista while also securing an option on KalVista’s lead drug KVD001 for diabetic macular edema. https://endpts.com/giant-merck-buddies-up-with-little-kalvista-on-lead-drug-igniting-stock-with-a-760m-deal
AbbVie has secured an option to buy Turnstone Biologics’ lead therapy combining an oncolytic virus approach and cancer vaccine. https://endpts.com/abbvie-jumps-on-the-oncolytics-bandwagon-embracing-turnstone-in-new-collaboration
Kaleido Biosciences and CoreBiome have formed a partnership to accelerate microbiome therapeutic development. http://www.biospace.com/News/kaleido-biosciences-and-corebiome-partner-to/472146?type=email&source=DD_101017
Lupin Inc., the U.S. subsidiary of India-based Lupin, plunked down $150 million to acquire New Jersey-based Symbiomix Therapeutics and its recently approved bacterial vaginosis treatment. http://www.biospace.com/News/lupin-coughs-up-150-million-cash-for-this-new/472218?type=email&source=GP_101117
Eight months after Trevena reported mixed Phase III results for its new pain therapy, they announced this week that they will be cutting 30% of its total workforce (21 staffers): https://endpts.com/battered-by-mixed-phiii-data-trevena-axes-rd-staff-and-hunkers-down-around-lead-pain-drug/
Karyopharm Therapeutics has entered into a $190 million deal with Ono for its late-stage cancer therapy selinexor and a follow-up compound it has in the pipeline. https://endpts.com/karyopharm-bags-a-193m-asian-deal-for-selinexor-and-followup-drug
OptiNose has taken advantage of a red-hot biotech IPO market to raise $120 million this week https://endpts.com/optinose-joins-the-biotech-ipo-fiesta-raises-120m-in-upsized-offering/
This is a weekly recap of selected industry news stories. You can read other additional blogs, see our recent job postings and learn more about us at www.dennispartners.com