COVID-19 has been all-consuming in 2020, grabbing the lion’s share of health news headlines. And while the FDA has made some of its own headlines during the pandemic—doling out guidance and approving the first treatment for COVID-19—it’s also been quietly tackling other health threats and challenges unrelated to the coronavirus, but nonetheless important. In October, for instance, the agency approved a mixture of three monoclonal antibodies to treat Ebola virus—it’s the first-ever FDA-approved treatment for the highly infectious and deadly disease that has hit Africa especially hard, and was touted as an important international collaboration. This year, in addition to approving new drugs for new indications, the FDA has also expanded indications of previously approved drugs. Here are five notable examples, including one that deals with cannabis. Ibrutinib plus rituximab In April 2020, the FDA expanded the indications for ibrutinib (Imbruvica) to include pairing with rituximab (Rituxan, Truxima) for the initial treatment of adult patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). The approval was based on results of an open-label, randomized controlled trial comparing the administration of ibrutinib plus rituximab to the chemotherapy regimen fludarabine, cyclophosphamide, and rituximab (FCR) in 529 adult patients aged 70 years or younger, with previously untreated CLL or SLL needing systemic therapy. The researchers found that progression-free survival (PFS) was enhanced in those receiving ibrutinib with rituximab (HR 0.34; 95% CI: 0.22, 0.52; p<0.0001). Of note, median PFS was not attained in either arm after a median follow-up of 37 months. Ibrutinib was initially approved for treating CLL, SLL, and mantle cell lymphoma, among other disorders. Rituximab was initially approved to treat non-Hodgkin's lymphoma, CLL, granulomatosis with polyangiitis, and microscopic polyangiitis. Olaparib plus bevacizumab In May 2020, the FDA expanded indications for the drug olaparib (Lynparza) when combined with bevacizumab (Avastin) as first-line maintenance treatment for adults with advanced epithelial ovarian, fallopian tube, or primary peritoneal cancer. Notably, patients treated with this drug must be in complete or partial response to first-line platinum-based chemotherapy with homologous recombination deficiency (HRD) positive status (ie, harbor either a deleterious or suspected deleterious BRCA mutation, and/or genomic instability). In clinical trials, the estimated median PFS in a sample of 387 patients with HRD-positive tumors was 37.2 months in the olaparib with bevacizumab arm vs 17.7 months in the placebo plus bevacizumab arm (HR 0.33; 95% CI: 0.25-0.45). Olaparib was initially approved for the treatment of ovarian cancer, breast cancer, pancreatic cancer, and prostate cancer. The FDA initially approved bevacizumab for colorectal cancer, non-small cell lung cancer, cervical cancer, and other cancers. Dapagliflozin Also in May, the FDA approved oral tablets of the type 2 diabetic medication dapagliflozin (Farxiga) for the treatment of adults with heart failure who exhibit reduced ejection fraction, as a means to curb risk of cardiovascular death and hospitalization secondary to heart failure. Dapagliflozin is the first sodium-glucose co-transporter 2 (SGLT2) inhibitor to garner approval for the treatment of New York Heart Association’s functional class II-IV heart failure with reduced ejection fraction. In a large clinical trial, investigators assigned patients to receive a once-daily dose of either 10 mg of dapagliflozin or placebo, and found that those taking dapagliflozin experienced fewer cardiovascular deaths, hospitalizations for heart failure, and acute heart-failure presentations vs those taking placebo. “Heart failure is a serious health condition that contributes to one in eight deaths in the US and impacts nearly 6.5 million Americans. This approval provides patients with heart failure with reduced ejection fraction an additional treatment option that can improve survival and reduce the need for hospitalization,” said Norman Stockbridge, MD, PhD, director of the Division of Cardiology and Nephrology in the FDA’s Center for Drug Evaluation and Research, in a press release. Cannabidiol oral solution In July 2020, the FDA approved a cannabidiol (CBD) oral solution (Epidiolex) for the treatment of seizures secondary to tuberous sclerosis complex (TSC) in patients aged 1 year and older. Of note, TSC is a genetic disorder leading to tumor growth in the central nervous system, along with the kidneys, lungs, and skin, thus resulting in seizures, developmental delay, and behavioral problems. Previously, CBD oral solution was approved to treat two rare forms of epilepsy, Lennox-Gastaut syndrome and Dravet syndrome, and is the only FDA-approved drug containing a cannabis-derivative. “The FDA continues to believe the drug approval process represents the best way to make new medicines, including any drugs derived from cannabis, available to patients in need of appropriate medical therapy such as the treatment of seizures associated with these rare conditions. This paradigm ensures new therapies are safe, effective, and manufactured to a high quality that provides uniform and reliable dosing for patients,” stated Douglas Throckmorton, MD, deputy center director for regulatory programs in the FDA’s Center for Drug Evaluation and Research, in a press release. Nivolumab plus ipilimumab In October of this year, the FDA approved the combination of nivolumab (Opdivo) and ipilimumab (Yervoy) as first-line treatment for patients with unresectable malignant pleural mesothelioma, the second FDA-approved treatment for this condition and first such approval in 16 years. Per the results of a randomized trial, patients receiving the experimental drug combo survived a median of 18.1 months vs 14.1 months in those receiving chemo. “Today’s approval of nivolumab plus ipilimumab provides a new treatment that has demonstrated an improvement in overall survival for patients with malignant pleural mesothelioma. In 2004, FDA approved pemetrexed in combination with cisplatin for this indication, and now patients now have an important, additional treatment option after more than a decade with only one FDA-approved drug regimen,” stated Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence, in a press release. Both nivolumab and ipilimumab were initially approved for treatment of numerous types of cancers including malignant pleural mesothelioma, melanoma, lung cancer, colon cancer, and others. Source
