The FDA’s Peripheral and Central Nervous System Drugs Advisory Committee has unanimously recommended approval of the New Drug Application for GW Pharmaceuticals’ proposed cannabidiol oral solution for the adjunctive treatment of seizures associated with 2 rare disorders, Lennox-Gastaut syndrome and Dravet syndrome, in patients 2 years and older.
The FDA’s Peripheral and Central Nervous System Drugs Advisory Committee has unanimously recommended approval of the New Drug Application for GW Pharmaceuticals’ proposed cannabidiol oral solution for the adjunctive treatment of seizures associated with 2 rare disorders, Lennox-Gastaut syndrome (LGS) and Dravet syndrome (DS), in patients aged 2 years and older.
LGS typically occurs between ages 3 and 5 years, can cause multiple seizure types, and is often resistant to anti-epileptic drugs. DS, a severe, infantile-onset, highly treatment-resistant epileptic encephalopathy, is associated with an elevated risk of premature death. Currently, there are no FDA-approved treatments for DS.
GW Pharmaceuticals’ product, which it plans to market as Epidiolex if it is eventually approved, would be both the first in a new category of anti-epileptic drugs and the first pharmaceutical formulated from plant-based cannabidiol. The exact mechanism of the anticonvulsant effect of cannabidiol is not known.
The advisory committee based its unanimous vote in favor of the drug’s benefit—risk profile on data from 3 randomized, double-blind, placebo-controlled trials, 2 of which were 14-week trials in patients with LGS. The third study was a 14-week trial in patients with DS. In all 3 studies, more patients who received cannabidiol had a 50% reduction in the number of seizures than did patients who received placebo.
Elizabeth Thiele, MD, PhD, director of pediatric epilepsy at Massachusetts General Hospital, professor of Neurology at Harvard Medical School, and a primary investigator for 1 of the trials in patients with LGS, said in a statement, “As a physician who treats LGS and [DS], I know that patients and their families usually face significant difficulties getting seizures under control using existing therapies. The results from these studies suggest that this pharmaceutical formulation of cannabidiol may provide hope for a new treatment option that may be effective for some patients.”
GW Pharmaceuticals says that it expects the drug to be approved by the FDA, on the strength of the advisory committee’s recommendation, on June 27, 2018, and to become commercially available in the second half of 2018.
Exagamglogene Autotemcel Meets End Points in Severe Sickle Cell Disease, β-Thalassemia
December 7th 2023Two posters set to be presented at the 65th American Society of Hematology Annual Meeting & Exposition met their primary and secondary end points regarding exagamglogene autotemcel therapy for sickle cell disease and β-thalassemia.
Read More
Oncology Onward: A Conversation With Dr Shereef Elnahal, Under Secretary for Health
April 20th 2023Shereef Elnahal, MD, MBA, under secretary for health at the Veterans Health Administration (VHA), sat for a conversation with our hosts Emeline Aviki, MD, MBA, Memorial Sloan Kettering Cancer Center, and Stephen Schleicher, MD, MBA, Tennessee Oncology, that covered the cancer footprint of the VHA.
Listen
NCCN Guidelines Update Adds Momelotinib Below Ruxolitinib for High-, Low-Risk Myelofibrosis
November 21st 2023Momelotinib was given category 2A and 2B status for patients with high- and low-risk myelofibrosis (MF) and MF with anemia. However, ruxolitinib retains a higher category of recommendation as a treatment for patients with MF.
Read More
Exploring Payer Coverage Decisions Following FDA Novel Drug Approvals
May 3rd 2022On this episode of Managed Care Cast, Ari D. Panzer, BS, lead author and researcher, then at Tufts Medical Center—now at Duke University—discusses the findings from his team’s investigation into coverage decisions by health plan insurers of the 66 drugs approved by the FDA in 2018.
Listen
Odevixibat Safe for Alagille Syndrome Based on Hepatic Changes
November 8th 2023Pooled phase 3 data presented at North American Society for Pediatric Gastroenterology, Hepatology & Nutrition 2023 support the benefit-risk profile of the ileal bile acid transport inhibitor in treating the rare liver disease.
Read More
Contributor: How Patients and Caregivers Can Be a Catalyst for Rare Disease Innovation
November 5th 2023Patient input and experiences play a crucial role in advancing rare disease research and therapy development, as they help define the disease, inform clinical trial design, and influence regulators and payers' decisions, ultimately serving as catalysts for innovation in the field.
Read More