This week the FDA granted Breakthrough Therapy status to three development programs and halted two different clinical trials. Here are the top regulatory news stories to follow this week:
Ocular Therapeutix, which had its lead drug rejected by the FDA last summer said that it remains on track to resubmit its application to the FDA for Dextenza (dexamethasone) for the treatment of pain after surgery. https://www.bizjournals.com/boston/news/2018/03/08/under-sec-scrutiny-bedford-s-ocular-therapeutix.html
The FDA granted Breakthrough Therapy designation to Galera Therapeutics’ GC4419 for reduction of the duration, incidence, and severity of severe oral mucositis induced by radiation therapy with or without systemic therapy. http://www.cancernetwork.com/palliative-and-supportive-care/antioral-mucositis-drug-granted-breakthrough-status
Proteostasis Therapeutics announced that the FDA has granted Breakthrough Therapy status and an Orphan Drug Designation for PTI-428, the Company’s cystic fibrosis transmembrane conductance regulator (CFTR) amplifier drug candidate. https://globenewswire.com/news-release/2018/03/12/1420865/0/en/Proteostasis-Therapeutics-Announces-FDA-Grants-Breakthrough-Therapy-Designation-for-PTI-428-in-Cystic-Fibrosis.html and https://globenewswire.com/news-release/2018/03/14/1422757/0/en/Proteostasis-Therapeutics-Announces-FDA-Grants-Orphan-Drug-Designation-for-PTI-428-in-Cystic-Fibrosis.html
The FDA granted Breakthrough Therapy Designation to Janssen and Astex Therapeutics’ erdafitinib for the treatment of metastatic urothelial cancer https://www.healio.com/hematology-oncology/genitourinary-cancer/news/online/%7B7b1f3cdb-f4be-4c75-9a40-49f68c1b68eb%7D/fda-grants-breakthrough-therapy-designation-to-erdafitinib-for-metastatic-urothelial-carcinoma
Sarepta has wrapped up talks with the FDA on how it should move forward with its new Duchenne muscular dystrophy drug: golodirsen. With notes from the FDA in hand, the company says it plans to seek rapid approval, with an NDA filed by the end of this year. https://endpts.com/sarepta-plays-nice-with-the-fda-sets-out-plans-for-second-duchenne-approval-by-years-end/?utm
Tetraphase announced that the FDA has accepted for filing of its NDA submission for eravacycline for the treatment of complicated intra-abdominal infections https://www.drugs.com/nda/eravacycline_180227.html
Advaxis disclosed that a Phase 1/2 trial of its immunotherapy drug axalimogene filolisbac, also known as axal, in combination with AstraZeneca plc’s Imfinzi has been placed on clinical hold by the FDA. https://www.biopharmadive.com/news/advaxis-clinical-hold-patient-death-axal/519007/
Solid Biosciences said that the FDA had halted a trial of its gene therapy treatment for Duchenne muscular dystrophy after one patient was hospitalized. https://www.bizjournals.com/boston/news/2018/03/14/shares-of-solid-biosciences-crater-as-fda-halts.html?ana=e_bost_bn_breakingnews&u=ko7ZzeXq4Nc5tasOqYHeeg0a5d9adc&t=1521123176&j=80504651
To better inform its regulation of biopharma advertising and promotion, the FDA has laid out plans for upcoming research into several advertising and promotional issues. https://www.raps.org/news-and-articles/news-articles/2018/3/fda-lays-out-plans-for-future-research-on-pharmace?utm
The FDA finalized ICH’s guidance on DNA-reactive substances that could potentially cause damage when present at low levels and potentially cause cancer. https://www.raps.org/news-and-articles/news-articles/2018/3/fda-finalizes-ich-m7-guidance-on-mutagenic-carcino?utm
After about five years as a standalone agency, the China Food and Drug Administration (CFDA) will merge into a gigantic national market supervision administration, with drug regulation as a new, second-tier bureau underneath. https://www.fiercepharma.com/pharma-asia/china-creates-new-drug-regulator-biggest-government-overhaul-years?mkt
In other news…
Orexigen Therapeutics said on Monday it filed for Chapter 11 bankruptcy protection and will also file a motion to pursue an auction and sale process of substantially all its assets. https://www.reuters.com/article/us-orexigen-bankruptcy/u-s-drugmaker-orexigen-files-for-chapter-11-bankruptcy-idUSKCN1GO0GU
Alnylam Pharmaceuticals announced that Sanofi Genzyme has declined its opt-in for the development and commercialization of lumasiran (ALN-GO1), an investigational RNAi therapeutic for the treatment of Primary Hyperoxaluria Type 1 (PH1). http://investors.alnylam.com/news-releases/news-release-details/alnylam-retains-global-rights-lumasiran-investigational-rnai
Pfizer has sold its early stage schizophrenia drug PF-04958242 to Biogen for a reported $590 million. https://endpts.com/biogen-is-hustling-a-schizophrenia-drug-into-phiib-after-pfizer-lets-it-go-in-590m-cns-fire-sale/?utm
AveXis and Genethon announced they have entered into an exclusive, worldwide license agreement for in vivo gene therapy delivery of AAV9 vector into the central nervous system for the treatment of spinal muscular atrophy. https://globenewswire.com/news-release/2018/03/13/1421661/0/en/AveXis-Enters-into-Licensing-Agreement-with-Genethon.html
Lundbeck announced that it will acquire Prexton Therapeutics and take over Prexton’s experimental asset foliglurax, which is being investigated as a supportive therapy for the treatment of the symptoms of Parkinson’s disease. https://www.biopharmadive.com/news/lundbeck-bulks-up-neuro-pipeline-with-prexton-buy/519334/
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