In 2018, the US Food and Drugs Administration approved 59 new drugs for medical use. This marks the highest record of approvals to date. This is in comparison to the 46 approvals in 2017 and 22 in 2016. The Administration’s Center for Drug Evaluation and Research (CDER) releases lists of recently authorized products annually, ranging from never before used in clinical practice to biosimilars
Here are some innovative products from last year.
Arikayce is the first to be certified under Congress-established Limited Population Pathway for Antibacterial and Antifungal Drugs (LPAD). Otherwise known as amikacin liposome inhalation suspension, Arikayce is intended for nontuberculous mycobacterial lung disease. However, usage is limited to infected patients who don’t respond to conventional treatment.
For approval, the FDA conducted a controlled clinical trial to evaluate Arikayce’s safety and efficacy. Results showed 29% of patients administered with the drug had no mycobacterial growth in their sputum cultures.
Tpoxx or tecovirimat is a first for smallpox medication. FDA Commissioner Dr. Scott Gottlieb states that the medicine was developed to “address the risk of bioterrorism” should the virus be used as a bioweapon.
Guided by the FDA’s Animal Rule, Tpoxx was given to infected animals to test its safety and efficacy. On healthy humans, the most frequent side effects noted were headaches, nausea and abdominal pains.
Trogarzo (ibalizumab-uiyk) is a new type of antiretroviral agent for multidrug-resistant (MDR) HIV-1. Most patients living with HIV respond well to a combination of antiretroviral therapy (ART) but a small percentage of people have developed resistance.
“Trogarzo is the first drug in a new class of antiretroviral medications that can provide significant benefit to patients who have run out of HIV treatment options,” says Dr. Jeff Murray, Deputy Director of the Division of Antiviral Products in the CDER.
Tested MDR patients exhibited a decrease in HIV-RNA virus in their blood one week after adding Trogarzo to their regimens. Twenty-four weeks after, 43% of the participants achieved HIV-RNA suppression.
Epidiolex is the pioneering marijuana-based medicine. It contains highly purified cannabidiol (CBD) without the psychoactive effects of tetrahydrocannabinol (THC).
The solution is indicated for seizures associated with Lennox-Gestaut Syndrome (LGS) and Dravet Syndrome (DS). This innovation spurs more research and development of potent cannabis drugs. Dr. Gottlieb reiterates the FDA’s commitment to take immediate action against the proliferation of CBD-containing products “with serious, unproven medical claims.”
This is the first validated therapy for the rare blastic plasmacytoid dendritic cell neoplasm (BPDCN). Prior to the approval, there was an urgent need for alternative options to intensive chemotherapy and bone marrow transplant. “Many patients with BPDCN are unable to tolerate this intensive therapy,” says Dr. Richard Pazdur, Director of the FDA’s Oncology Center of Excellence.
The infusion contains tagraxofusp-erzs as an active ingredient and can be used on adult and pediatric patients. Trials involved two cohorts: patients with untreated BPDCN and those with relapse or refractory BPDCN. Results showed 54% of the participants in the first group achieved complete remission (CR). The other group achieved only one CR and another with a skin abnormality not indicative of active disease (CRc).
Crysvita (burosumab-twza) became the first indicated for x-linked hypophosphatemia (XLH) in adults and children. Dr. Julie Beitz explains that this form of rickets render vitamin D therapy ineffective. She is the Director of the Office of Drug Evaluation III in the CDER.
Scientific investigations showed 94% of adults and almost 100% of children achieved normal phosphorus levels after receiving Crysvita. Their X-ray findings also improved. Thus, it was granted Breakthrough Therapy and received Orphan Drug designations by the FDA.
Further discoveries and future for research
Also included on the list are Aimovig (erenumab-aooe) for migraine-prevention, Ultomiris (ravulizumab-cwvz) for adults with paroxysmal nocturnal hemoglobinuria (PNH), and Braftovi (encorafenib) for unresectable or metastatic melanoma.
The availability of novel therapeutic and biological products entails improved medical options. Pharmaceutical companies and the FDA continue to collaborate in creating and testing such outputs to cure rare and severe illnesses known today. All these in the efforts to eradicate every disease known to man.