Fast Track/Breakthrough Designations
Sanofi and Regeneron Pharmaceuticals received FDA Breakthrough Therapy designation for cemiplimab (REGN2810) for the treatment of metastatic cutaneous squamous cell carcinoma (CSCC) and locally advanced and unresectable CSCC, the second deadliest skin cancer after melanoma. Cemiplimab is an investigational human, monoclonal antibody targeting PD-1. 
            The company previously reported positive, preliminary results for cemiplimab from two cohorts involving 26 advanced CSCC patients in a Phase I study of nearly 400 patients. A Phase II, single-arm, open label trial of cemiplimab is currently enrolling patients for metastatic CSCC and locally advanced and unresectable CSCC. Cemiplimab is being jointly developed by Sanofi and Regeneron under a global collaboration. Pending data results, the companies anticipate submitting a biologics license application for cemiplimab with the FDA in 1Q18.
            CSCC is the second most common type of skin cancer in the U.S. Although CSCC has a good prognosis when caught early, it can prove especially difficult to treat when it progresses to advanced stages.
Breakthrough Therapy designation serves to expedite the development and review of drugs that target serious or life-threatening conditions.
 
Audentes Therapeutics’ AT132, the company’s gene therapy candidate being developed to treat X-Linked Myotubular Myopathy (XLMTM), was granted Rare Pediatric Disease and Fast Track designations. AT132 also received Orphan Drug designation from both the FDA and European Medicines Agency.
            XLMTM is a rare monogenic disease characterized by extreme muscle weakness, respiratory failure and early death, with an estimated 50% mortality rate by 18 months of age.
            Audentes recently enrolled the first patient in a Phase 1/2 trial of AT132 in patients with XLMTM. Preliminary data from ASPIRO is expected to be available in 4Q17. The clinical development program for AT132 also includes a retrospective medical review of 112 male subjects. This analysis confirmed and expanded upon the understanding of the significant disease burden of XLMTM.  Audentes is also conducting a prospective natural history and run-in study.  The primary objectives are to characterize the clinical condition of children with XLMTM, identify subjects for potential enrollment and serve as a baseline for trials.
The Fast Track program facilitates the development and expedites the review of new drugs which show promise in treating a serious or life-threatening disease and address an unmet medical need. Drugs with this designation may qualify for priority review if relevant criteria are met.
 
Submissions
AbbVie in cooperation with Neurocrine Biosciences, has submitted a New Drug Application (NDA) to the U.S. FDA for elagolix, an investigational, orally administered gonadotropin-releasing hormone (GnRH) antagonist, being evaluated for the management of endometriosis with associated pain. In two Phase III studies, elagolix demonstrated superiority compared to placebo in reducing three types of endometriosis-associated pain – daily menstrual pelvic pain, non-menstrual pelvic pain and painful intercourse.          
            The NDA is supported by data from the largest prospective randomized endometriosis clinical trials conducted to date, which evaluated the safety and efficacy of elagolix in nearly 1,700 women with moderate-to-severe endometriosis-associated pain. The data demonstrated that, at month three and month six, both elagolix doses resulted in a statistically significant higher proportion of responders for menstrual pain (dysmenorrhea) and non-menstrual pelvic pain associated with endometriosis versus placebo. Significant improvements compared to placebo were also observed for painful intercourse (dyspareunia).
 
Sunovion Pharmaceuticals has submitted a New Drug Application (NDA) to the U.S. FDA for dasotraline, an investigational, dual-acting dopamine and norepinephrine reuptake inhibitor (DNRI), for the treatment of children, adolescents and adults with attention deficit hyperactivity disorder (ADHD). Dasotraline’s pharmacokinetic properties, such as an extended half-life, and clinical study findings support its potential for sustained control of ADHD symptoms over the 24-hour dosing interval with once-daily dosing.
            The NDA submission is supported by data from the clinical program for dasotraline in ADHD, which included four placebo-controlled safety and efficacy studies, as well as two long-term studies that assessed the safety of dasotraline in patients with ADHD for up to one year. In total, approximately 2,500 patients with ADHD were evaluated in these studies utilizing dasotraline dosages in the range of 2 mg/day to 8 mg/day. Dasotraline was generally well tolerated.
Dasotraline is also being investigated for the treatment of binge eating disorder (BED) in adults.
 
Akcea Therapeutics, Inc., an affiliate of Ionis Pharmaceuticals, has submitted a NDA to the U.S. FDA for volanesorsen, an investigational medicine for the treatment of familial chylomicronemia syndrome (FCS).
            FCS is a severe, rare disorder characterized by extremely high levels of triglycerides, symptoms such as abdominal pain that affect daily living, and the risk of recurrent, potentially fatal, acute pancreatitis. People with FCS are unable to effectively metabolize large, triglyceride-rich lipid particles called chylomicrons due to a deficiency in lipoprotein lipase, an enzyme that helps to break down triglycerides. There is no effective therapy available.
            The APPROACH and COMPASS studies demonstrated significant reductions in triglyceride levels and reduced risk of pancreatitis.