FDA Actions
The FDA approved ocrelizumab to treat adult patients with relapsing forms of multiple sclerosis (MS) and primary progressive multiple sclerosis (PPMS). The drug is a monoclonal antibody with a new mechanism of action: targeting B cells. It is also the first drug approved for PPMS. Clinicians administer the drug by IV infusion every six months. Efficacy was shown in two trials that included more than 1,600 participants with relapsing forms of MS treated for 96 weeks using interferon beta-1a (Rebif) as a comparator. In both studies, the patients receiving ocrelizumab experienced reduced relapse rates (Trial 1: 0.16 vs. 0.29; 46% lower rate with ocrelizumab; P < 0.001. Trial 2: 0.16 vs. 0.29; 47% lower rate; P < 0.001). Ocrelizumab also reduced disability progression at 12 and 24 weeks and significantly reduced the mean number of gadolinium-enhancing lesions. The drug also was studied in 732 patients with PPMS; participants receiving ocrelizumab showed a longer time to the worsening of disability compared to placebo. Ocrelizumab comes with a medication guide that describes important information about the drug’s uses and risks. The drug may increase the risk for malignancies, especially breast cancer. Vaccination with live or live attenuated vaccines is not recommended in patients receiving ocrelizumab. The most common side effect was upper respiratory tract infection. The FDA granted this application breakthrough therapy designation, fast-track designation, and priority review. Ocrelizumab is marketed as Ocrevus. It will be priced at $65,000 per year, which is less than many existing MS drugs.
The FDA has approved dupilumab injection for the treatment of moderate-to-severe eczema (atopic dermatitis). The drug is a human monoclonal antibody that attaches to the IL-4 receptor alpha subunit. It is administered by subcutaneous injection. Safety and efficacy were established in three placebo-controlled trials of more than 2,100 adult participants with moderate-to-severe atopic dermatitis not adequately controlled by topical medications. Patients receiving dupilumab achieved greater response defined as clear or almost clear skin, and experienced a reduction in itch after 16 weeks of treatment. The drug can cause allergic reactions and eye problems, such as conjunctivitis and keratitis. The FDA granted the drug priority review and breakthrough therapy designation. Dupilumab is marketed as Dupixent. It is expected to cost $37,000 per year.
The FDA has approved safinamide as an add-on treatment for patients with Parkinson’s disease who are taking levodopa/carbidopa and experiencing “off” episodes (periods when the medication is less effective, resulting in decreased mobility and tremor). Safinamide exerts most of its effect by blocking monoamine oxidase similar to selegiline and rasagiline. However, unlike those drugs, the effect is reversible. Safinamide also inhibits glutamine release, blocks dopamine reuptake, and blocks sodium and calcium channels. Efficacy was shown in a clinical trials of some 1,200 patients taking levodopa in which “on” time was increased and motor function improved. Like all MAO inhibitors, there are multiple drug-drug interactions, including other MAOIs, opioids, St. John’s wort, SNRIs, TCAs, and others. Common side effects include uncontrolled involuntary movement, falls, nausea, and insomnia. Safinamide is marketed as Xadago. It is expected to cost $8,000 for a one-year supply.
The FDA has approved deflazacort to treat patients ≥ 5 years of age with Duchenne muscular dystrophy (DMD). The drug is an oral steroid that works to decrease inflammation. Although corticosteroids are used commonly to treat DMD, this is the first corticosteroid approved specifically for this indication. Also, it is the first approval of deflazacort for any indication in the United States. Effectiveness was shown in a trial of nearly 200 patients age 5-15 years. At 12 weeks, patients taking deflazacort demonstrated improvements in clinical assessment of muscle strength compared to placebo. The drug was granted fast-track designation, priority review, and orphan drug designation. Deflazacort is marketed as Emflaza. It is expected to cost $89,000 per year.
In this section: The agency approves exciting new treatment for multiple sclerosis, green lights an injection for atopic dermatitis, gives the go ahead to an add-on treatment for Parkinson's disease, and signs off on a drug for Duchenne muscular dystrophy.
Subscribe Now for Access
You have reached your article limit for the month. We hope you found our articles both enjoyable and insightful. For information on new subscriptions, product trials, alternative billing arrangements or group and site discounts please call 800-688-2421. We look forward to having you as a long-term member of the Relias Media community.