Over the course of 2017, the number of approvals granted by the US Food and Drug Administration (FDA) reached a 21-year high. More than 1,000 generic drugs were approved by the agency, according to FDA Commissioner Scott Gottlieb, MD, while first-time generic drug approvals numbered 74. Novel drug approvals (new drugs containing previously unused active ingredients) also increased, reaching a total of 46 - more than double the 22 to receive the green light in 2016.
With so many new medicines available in local US pharmacies, ModRN Health has picked out six of the most significant approvals granted by the FDA during 2017, including important new generic versions of commonly used medicines, novel drugs providing new therapy options for rare diseases, and treatments to help patients manage common conditions.
1. Ocrevus (ocrelizumab)
Approved in March 2017, Ocrevus is a new drug indicated to treat adult patients with relapsing forms of multiple sclerosis (MS) and primary progressive multiple sclerosis. The drug, developed by Roche subsidiary Genentech, is administered via an intravenous infusion from a healthcare professional.
MS is a chronic, inflammatory autoimmune disease of the central nervous system. Patients with the condition often experience episodes where function worsens, followed by remission periods. It is a debilitating condition and patients often deteriorate over time.
Clinical trials demonstrated patients given Ocrevus had reduced relapse rates and reduced worsening of their condition. Among the common side effects of Ocrevus were upper respiratory tract infections, skin infections and lower respiratory tract infection.
2. Dupixent (dupilumab)
Another significant drug to receive the green light from the FDA in March last year was Dupixent (dupilumab), which is indicated for the treatment of moderate-to-severe eczema (atopic dermatitis). The Regeneron Pharmaceuticals medication received Priority Review and Breakthrough Therapy designation from the FDA.
Dupixent is administered as a subcutaneous injection (injection under the skin) and is intended for use in patients whose eczema is not adequately controlled by topical therapies. It can be used with or without topical corticosteroids and is particularly useful in treating patients for whom topical therapies are not advisable.
Among the possible side effects of Dupixent are serious allergic reactions and ocular issues, such as pink eye or inflammation of the cornea. The most common reactions were injection site reactions; cold sores on the mouth or lips; and eye or eyelid inflammation, which may include redness, swelling, and itching.
3. Radicava (edaravone)
In May 2017, Mitsubishi Tanabe Pharma America was granted approval for Radicava (edaravone) as a treatment for amyotrophic lateral sclerosis (ALS). The FDA granted Radicava orphan drug designation, which is designed to encourage the development of drugs to treat rare diseases.
ALS is a rare progressive condition which attacks nerve cells responsible for controlling voluntary muscles, for example, those that control chewing, walking, breathing and talking. Between 12,000 and 15,000 people in the United States suffer from the condition, according to figures from the Centers for Disease Control and Prevention.
Radicava is administered via an intravenous infusion given by a healthcare professional. Clinical trials demonstrated the drug slowed the decline of daily function in patients when compared to patients given a placebo. Common adverse reactions to Radicava include bruising and gait disturbance. It is also associated with allergic reactions to sodium bisulfite (one of the ingredients of the drug), as well as hives, swelling, and shortness of breath.
4. Generic Truvada (emtricitabine, tenofovir, disoproxil fumarate)
June 2017 saw Teva Pharmaceuticals granted approval for its generic version of the HIV combination treatment Truvada. In addition to providing treatment for HIV-1 in combination with other antiretroviral agents, the drug may also be used for pre-exposure prophylaxis to prevent sexually-acquired HIV infection in high-risk adults.
It is not yet known when the drug will be available in the United States, but when it appears in US pharmacies it is expected to be significantly cheaper than the branded original drug manufactured by Gilead Sciences.
Common side effects of Truvada include mild diarrhea, mild nausea or stomach pain, headache, dizziness, depressed mood, mild itching, skin rash, unusual dreams, and changes to the shape or location of body fat.
5. Kymriah (tisagenlecleucel)
In August 2017, the FDA made one of its most significant approvals of the year, making the first cell-based gene therapy available in the United States. Kymriah (tisagenlecleucel) is used to treat patients with acute lymphoblastic leukemia (ALL) - a form of cancer - which has not been cured or has returned following chemotherapy.
The Novartis drug is the first CAR-T therapy available to cancer patients in the United States and was granted Priority Review and Breakthrough Therapy designations by the FDA. In December, the FDA also approved Luxturna (voretigene neparvovec-rzyl), a drug used to treat a rare form of inherited vision loss and blindness, and the second gene therapy available in the United States.
Each dose of Kymriah is a customized treatment using T-cells from the individual patient. It is approved for adults, young adults, and pediatric patients. The drug has the potential to cause a range of severe side effects, including cytokine release syndrome, serious infection, low blood pressure, acute kidney injury, fever, and decreased oxygen.
6. Trelegy Ellipta (fluticasone furoate, umeclidinium, vilanterol inhaler)
The Trelegy Ellipta (fluticasone furoate, umeclidinium, vilanterol inhaler) from GlaxoSmithKline was granted FDA approval in September 2017. It is indicated for maintenance treatment of patients with chronic obstructive pulmonary disease (COPD).
The product is the first once-daily treatment combining three active ingredients - a corticosteroid, an anticholinergic, and a LABA medication - in a single inhaler. It is not indicated for treatment of asthma or acute bronchospasm. The Trelegy Ellipta is now available in pharmacies across the United States.
The most common adverse reactions experienced by patients using the Trelegy Ellipta are headaches, back pain, a distortion of taste, diarrhea, cough, throat pain and gastroenteritis.
