Top Regulatory News Stories – Week Ending June 23, 2017

This has been a busy week with a few big announcements from the FDA, the approval of one NME, and several other program approvals.  Here are a few of the top stories for regulatory professionals to follow:

FDA Commissioner Scott Gottlieb vowed to eliminate the backlog of orphan drug designation requests and said he will soon release “modern and risk-based” tools for assessing new treatments, especially for rare diseases and conditions with no effective treatments. Full Story Here

FDA senior policy adviser Ritu Nalubola explained to attendees at the DIA annual conference in Chicago that the agency is in the early stages of building capacity to regulate treatments that use CRISPR-Cas9 technology, but he did not provide any details: Full Story Here

Also at the DIA conference Peter Marks, Director of CBER, reported that there have been 19 requests for the new RMAT designation (Regenerative Medicine Advanced Therapy) since January and four have been granted: Full Story Here

Axovant Sciences’ investigational drug nelotanserin has secured fast track designation from the FDA for the treatment of visual hallucinations disorder in dementia with Lewy bodies. Full Story Here

Melinta Therapeutics announced that the FDA has approved Baxdela (delafloxacin) for the treatment of acute bacterial skin and skin structure infections in adults Full Story Here

The FDA granted approval of Haegarda to CSL Behring for the prevention of Hereditary Angioedema attacks in adolescent & adult patients. Haegarda is a human plasma-derived, purified, pasteurized, freeze-dried concentrate Full Story Here

The FDA approved Adamis Pharmaceuticals’ Epinephrine Injection for the emergency treatment of allergic reactions including anaphylaxis. This could provide consumers with an alternative to Mylan’s epinephrine autoinjector, EpiPen. Full Story Here

The FDA approved Neos Therapeutics’ controlled-release version of an old ADHD therapy, methylphenidate. This tablet is designed to deliver the medication in a gradual manner over time, instead of all at once.  Full Story Here

Shire reported that the FDA has approved Mydayis, a once-daily treatment comprised of three different types of drug-releasing beads for patients aged 13 years and older with ADHD. Full Story Here

Ipsen announced that the FDA has expanded the approved use of Dysport (abobotulinumtoxinA) for the treatment of lower limb spasticity in adults based on its sBLA. Dysport was approved for the treatment of upper limb spasticity in adults in 2015 and it was approved to treat pediatric patients with lower limb spasticity in 2016. Full Story Here

The FDA granted orphan drug designation for ResQ Pharma’s LipidRescue Therapy for the indication of local anesthetic systemic toxicity. Full Story Here

The FDA also granted Orphan Drug Designation to biotech startup Ammonett Pharma’s orally-administered mini-pill Growth Hormone for the treatment of Growth Hormone Deficiency. Full Story Here 

Novartis subsidiary Sandoz has secured approval from the European Commission for its biosimilar Rixathon to treat blood cancers and immunological diseases. With this latest approval, Sandoz currently has four biosimilar medicines approved in Europe. Full Story Here

Seattle Genetics submitted an sBLA to FDA for ADCETRIS (Brentuximab Vedotin) for the treatment of Cutaneous T-Cell Lymphoma http://www.businesswire.com/news/home/20170620005551/en/Seattle-Genetics-Submits-Supplemental-Biologics-License-Application This comes a day after the company suspended its phase 3 acute myeloid leukemia trial of vadastuximab talirine due to a higher rate of death incidents.  http://clinicaltrials.pharmaceutical-business-review.com/news/seattle-genetics-scraps-leukemia-drug-trial-over-safety-concerns-200617-5847727

The FDA has accepted Rigel Pharmaceutical’s NDA for Tavalisse (fostamatinib disodium) in patients with chronic or persistent immune thrombocytopenia. Rigel expects the FDA to complete its review by April 17, 2018. Full Story Here

The FDA has issued a Complete Response Letter to Pfizer in connection with their biosimilar candidate for anemia. Full Story Here

In other industry news…

Merck disclosed that it is pausing enrollment in two studies of its checkpoint inhibitor Keytruda (pembrolizumab) due to patient deaths. http://www.biopharmadive.com/news/merck-keytruda-deaths-myeloma-pause-trial/444881/

CRO Parexel International has agreed to be acquired by Pamplona Capital Management in a $4.6 billion deal:  http://www.cnbc.com/2017/06/20/parexel-to-be-acquired-by-pamplona-capital.html

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