At first glance, the number of drugs that received accelerated approval from the U.S. FDA’s Center for Drug Evaluation and Research (CDER) in 2023 was nothing to write home about. Yes, CDER granted nine accelerated approvals last year, up from six in 2022. But the proportion of novel drugs with accelerated approval was 16% both years. And when compared with the 12 drugs in 2020 and the 14 that received accelerated approval in 2021, last year’s crop was a little lackluster. However, a deeper look at the 2023 class of accelerated approvals shows a historic milestone. For the first time since the path was created in 1992, the number of novel biologics getting accelerated approval at CDER outpaced the number of small-molecule drugs.
Patients with amyotrophic lateral sclerosis (ALS) have a median survival of 2 to 5 years. There are 3 FDA-approved drugs for ALS (riluzole, edaravone and Relyvrio [phenylbuturate/taurursodiol]), but they only lead to modest benefit. There are several pathways involved in the disease, but all of them lead to neuroinflammation.
In 2023, the BioWorld Neurological Diseases Index (BNDI) closed with a 4.36% increase, outperforming the Nasdaq Biotechnology Index (up 3.74%) but falling short of the surge seen in the Dow Jones Industrial Average (up 13.7%). The rebound marked a significant turnaround from November, when BNDI recorded an 18.37% year-to-date decline.
Sooma Oy recently raised €5 million (US$5.4 million) in funding to help the company expand access to its transcranial direct current stimulation device which allows patients to treat their depression at home. “This is a significant milestone for us that enables us to help more patients globally and help us develop the company to serve the unmet need,” Tuomas Neuvonen, Sooma's co-founder and CEO, told BioWorld.
Cytokinetics Inc. has disclosed cardiac myosin inhibitors reported to be useful for the treatment of Becker muscular dystrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, spinal cord injury, limb girdle muscular dystrophy, stroke, tendinitis and tremor, among others.
Jawaharlal Nehru Centre for Advanced Scientific Research has divulged naphthalene monoimide compounds acting as modulators of liquid-liquid phase separation (LLPS) of tau protein reported to be useful for the treatment of traumatic encephalopathy, progressive supranuclear palsy, tauopathies, frontotemporal dementia and Alzheimer’s disease.
Recent studies have identified HAVCR2, which encodes immune checkpoint molecule TIM-3 (T-cell immunoglobulin mucin receptor 3), as a risk gene for late-onset Alzheimer’s disease (AD).
Ono Pharmaceutical Co. Ltd. has entered into a collaboration agreement with Sibylla Biotech SpA to generate novel drug candidates for neurological disorders. Under this collaboration, Sibylla will utilize its proprietary Pharmacological Protein Inactivation by Folding Intermediates Targeting (PPI-FIT) protein degradation technology platform.
Kv1.3 is a voltage-gated potassium channel that plays a crucial role in neuroinflammation and neurodegeneration in Parkinson’s disease and other disorders. Preclinical studies have shown that Kv1.3 inhibition confers neuroprotection against neurodegenerative disorders.