This segment discusses the recent drug approvals from the US FDA in various severe diseases. The write-ups describe the role of a new drug in the treatment of serious conditions such as myeloma, optical disease, and rhinitis. The FDA is the strongest regulatory body in USA which takes care of health of the public by assuring the safety and effectiveness of the human drugs, medical devices and other products used in health care.
FDA approves new treatment for refractory multiple myeloma1
On July 3, 2019, the U.S. Food and Drug Administration (U.S. FDA) approved Xpovio (selinexor) tablets in combination with the corticosteroid dexamethasone for the treatment of adult patients with relapsed refractory multiple myeloma (RRMM) resistant to different forms of treatment. Prior to the drug selinexor, the treatment option available to a patient with relapsed refractory multiple myeloma only used to slow down the spread of cancer. It was a very exhaustive process. Multiple myeloma, also referred to as plasma cell myeloma, is a cancer that begins in plasma cells (white blood cells that produce antibodies).
Xpovio is an orally available, small-molecule inhibitor of CRM1 (chromosome region maintenance 1 protein, exportin 1 or XPO1), with potential antineoplastic activity. Efficacy of the drug was studied in 83 patients with RRMM treated with Xpovio in combination with dexamethasone, which was effective in treating serious conditions. The FDA granted the approval of Xpovio to Karyopharm Therapeutics.
FDA warns re-packers of API for violating Good Manufacturing Practice2
On July 2, 2019, U.S. Food and Drug Administration (U.S. FDA) issued warning letters to three re-packers of active pharmaceutical ingredients (API) - B&B Pharmaceuticals, Inc. and Asclemed USA, Inc., doing business as Enovachem and Spectrum Laboratory Products, Inc., for significant violations of current good manufacturing practice (CGMP) requirements. U.S. market is the safest market as it has strict regulatory guidelines and protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. However, due to globalization and unauthorized deals in the pharmaceutical sector, there is an increasing threat to the quality of drug manufacturing.
FDA approves the first treatment of neuromyelitis optica spectrum disorder (NMOSD)3
On June 27, 2019, the US FDA approved Soliris (eculizumab) injection for intravenous use for the treatment of neuromyelitis optica spectrum disorder (NMOSD), a rare autoimmune disease of the central nervous system in adult patients who are anti-aquaporin-4 (AQP4) antibody positive. In patients with NMSOD, the body's immune system mistakenly attacks healthy cells and proteins in the body, most often in the optic nerves and spinal cord resulting into the attack of optic neuritis, eye pain and vision loss. Soliris is a monoclonal antibody that targets complement protein C5, preventing cleavage to C5a and C5b, and the formation of the terminal complement complex C5b-9. Inhibition of this complex prevents complement mediated intravascular hemolysis in paroxysmal nocturnal hemoglobunuria, complement mediated microangiopathy in atypical hemolytic uremic syndrome, and immune mediated inflammation and damage of the central nervous system in neuromyelitis optica spectrum disorder. The efficacy of the drug was studied in a clinical study of 143 patients with NMOSD who had antibodies against AQP4 (anti-AQP4 positive) who were randomized to receive either Soliris treatment or placebo. Compared to treatment with placebo, the study showed that treatment with Soliris reduced the number of NMOSD relapses by 94 percent over the 48-week course of the trial. The FDA granted the approval of Soliris to Alexion Pharmaceuticals.
FDA approves the first treatment for chronic rhinosinusitis with nasal polyps4
On June 26, 2019, US FDA approved Dupixent (dupilumab) to treat adults with nasal polyps (growths on the inner lining of the sinuses) accompanied by chronic rhinosinusitis (prolonged inflammation of the sinuses and nasal cavity). Nasal polyps is a disease which can lead to loss of smell which can often require surgery to remove the polyps. Dupixent Dupilumab is a recombinant human IgG4 antibody to the IL-4 receptor that works by inhibiting the activation of certain pro-inflammatory cytokines that are implicated in the pathophysiology of several allergic and atopic conditions, including asthma, chronic rhinosinusitis with nasal polyps, and food and environmental allergies. The efficacy and safety of Dupixent were established in two studies with 724 patients, 18 years and older with chronic rhinosinusitis with nasal polyps who were symptomatic despite taking intranasal corticosteroids.
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