new drug application

Osmotica Pharmaceuticals has submitted an amended new drug application (NDA) to the U.S. Food and Drug Administration (FDA) requesting the approval of arbaclofen extended release (ER) tablets for the treatment of spasticity in people with multiple sclerosis (MS). Arbaclofen ER (brand name…

Janssen has submitted an application to the U.S. Food and Drug Administration (FDA) asking for ponesimod to be approved as an oral treatment for adults with relapsing multiple sclerosis (MS). Ponesimod, formerly ACT-128800, is an experimental treatment that targets the protein sphingosine-1-phosphate…

An application has been submitted to approve ozanimod as an oral treatment for adults with relapsing forms of multiple sclerosis in the U.S., according to its developer, Celgene. “New oral treatment options with differentiated profiles like ozanimod are needed to help address an unmet need for people with relapsing forms of MS,” Jay Backstrom, MD, Celgene’s chief medical officer, said in a press release. Celgene's New Drug Application has been submitted to the U.S. Food and Drug Administration. Earlier this month, the company submitted a marketing authorization application to the European Medicines Agency covering the treatment of adults with relapsing-remitting MS. “With concurrent applications in the U.S. and EU, we look forward to advancing this promising medicine through the regulatory review process to provide a new option for the treatment of (relapsing MS) in 2020,” Backstrom said. Ozanimod is designed to cause the retention of immune cells in lymphoid tissues, thereby blocking their migration to the central nervous system — brain and spinal cord — and preventing damage to nerve fibers and their protective layer, called myelin. The investigational therapy selectively binds to S1P receptor subtypes S1P1 and S1P5. The NDA application is based on positive findings from two multicenter, double-blind, Phase 3 trials called SUNBEAM and RADIANCE part B. Both studies demonstrated that ozanimod reduced the number of relapses and brain lesions. In the SUNBEAM Phase 3 trial, 1,346 participants with relapsing MS were randomized to one daily dose of 0.92 or 0.46 mg of ozanimod — equivalent to 1 mg and 0.5 mg of the therapy’s HCI formulation — or Avonex (interferon beta-1a, marketed by Biogen) for at least 12 months. Results showed that treatment with ozanimod led to fewer relapses and brain lesions, as well as clinically meaningful improvements in processing speed compared with Avonex. In the Phase 2/3 RADIANCE trial, patients were divided in two parts: in part A, participants received either one daily dose of ozanimod (0.5 mg or 1.0 mg) or a placebo for 24 weeks; in part B, a 96-week open-label extension study completed by 223 patients, those initially on placebo switched to ozanimod. As in the SUNBEAM trial, results of part A of the RADIANCE trial revealed a reduction in the number of brain lesions from weeks 12 to 24, as well as less frequent relapses compared with a placebo. Treatment with ozanimod was safe and well-tolerated. Findings of part B of the study included an increased percentage of patients free of T1 lesions on MRI (magnetic resonance imaging) scans — which refer to areas of active inflammation and disease activity — after two years of treatment, from 58.5–69.0% of patients in part A to 86.5–94.6% of patients in part B. T2 lesions, a measure of the total amount of MRI lesions — both old and new — and relapse rate remained low in patients maintained on ozanimod (more significantly with the higher dose of 1.0 mg), and dropped in those who switched from a placebo. The scientists also analyzed ozanimod’s benefits using data from the SUNBEAM and RADIANCE part B trials, which covered 2,659 patients treated over one to two years. Compared with Avonex, ozanimod reduced the annualized relapse rates — the number of relapses per year — by 42% in the higher dose group and 26% in the lower dose group. Treatment with ozanimod also lessened the relapse rate requiring steroid treatment or hospitalization by 43% (in the 1 mg dose group) and 26% (in the 0.5 mg dose group) compared with Avonex treatment. In addition to MS, ozanimod is also being developed for patients with ulcerative colitis and Crohn's disease, two inflammatory bowel diseases.

The U.S. Food and Drug Administration (FDA) has agreed to review Alkermes’ request to approve diroximel fumarate (BIIB098) as a treatment for relapsing forms of multiple sclerosis (MS), the company announced. A final decision by the FDA is expected in the fourth quarter of 2019. If approved, diroximel…

Novartis is seeking U.S. and European approval of its investigational oral agent siponimod to treat adults with secondary progressive multiple sclerosis (SPMS). The U.S. Food and Drug Administration (FDA) has accepted for review the company’s New Drug Application, while the European Medicines Agency (EMA) has accepted for review…

Seven years after it first denied the request, the U.S. Food and Drug Administration accepted for review EMD Serono’s resubmitted New Drug Application (NDA) for cladribine tablets (brand name Mavenclad) as a treatment for patients with relapsing forms of multiple sclerosis (MS). The regulatory agency agreed EMD…

The U.S. Food and Drug Administration has asked Celgene to add more information to its New Drug Application for ozanimod as a treatment for relapsing forms of multiple sclerosis. Celgene said it plans to meet with FDA officials to make sure it understands exactly what new information the agency…