Lundbeck Pharmaceuticals Ltd.

Infantile spasms is a rare form of epilepsy. Credit: Shutterstock/FAMILY STOCK. The US Food and Drug Administration (FDA) has signed off on a ready-to-use formulation of vigabatrin developed by Pryos Pharmaceuticals for infantile spasms, marking the first approval for a novel drug formulation in this space in 15 years. US-based Pyros has developed a ready-to-use oral solution of vigabatrin, named Vigafyde. The drug is indicated for paediatric patients with infantile spasms aged one month to two years where the potential benefits outweigh the potential risk of vision loss, according to a 17 June press release. Infantile spasms, a rare form of epilepsy , is currently treated in the US with Lundbeck Pharmaceuticals’ Sabril (vigabatrin) and its generic versions, the former being approved by the FDA in 2009. Lundbeck gained US commercialisation rights to the drug from Sanofi . Sabril is available as 500mg tablets and 500mg powder for oral solution, as per its drug label . Vigabatrin currently has an FDA Risk Evaluation and Mitigation Strategy (REMS) Programme due to the drug’s known risk of causing permanent vision loss. Those eligible to take Vigafyde must be enrolled in the Vigabatrin REMS. Pryos will roll Vigafyde out with the REMS programme in H2 2024. The company also has a personalised support team on hand to assist families through the treatment journey. See Also: Johnson & Johnson submits BLA to FDA for lung cancer drug FDA approves MSD’s KEYTRUDA for advanced endometrial cancer Vigafyde will join Pyros’ other vigabatrin drug Vigpoder, an oral solution, which is therapeutically equivalent to Sabril, and was approved by the FDA in 2022. Infantile spasms usually begin in children aged one year or less and can lead to long-term neurological conditions such as continued seizures, other forms of epilepsy and autism spectrum disorder. Vigafyde works by inhibiting GABA transaminase. This increases GABA levels in the brain and helps modulate neuronal excitability, giving better seizure control. Sabril saw peak US sales of $229m in 2017 but after several market exclusivities expired , sales slumped. It is estimated to generate just $12m in annual sales by 2030, according to GlobalData’s Pharma Intelligence Centre. GlobalData is the parent company of Pharmaceutical Technology . Pyros’ chief operating officer Edwin Urrutia said: “This is a momentous day for patients and caregivers who have long awaited the first ready-to-use vigabatrin.” Pyros also has two other drugs in its pipeline, PYR-001 and PYR-002, with undisclosed therapeutic targets. PYR-001 is in late-stage clinical trials whilst PYR-002 is still in the early stages of development.

SAN DIEGO--(BUSINESS WIRE)-- Actio Biosciences, Inc., a biotechnology company leveraging a novel platform approach to genetics and precision medicine to develop new therapeutics that target shared underlying biology in both rare and common diseases, today announced that Ben Cravatt, Ph.D., has joined the company’s board of directors. Dr. Cravatt is professor and Norton B. Gilula Chair of Chemical Biology in the Department of Chemistry at The Scripps Research Institute. His research group is interested in developing chemical proteomic technologies that enable protein and drug discovery on a global scale and applying these methods to characterize biochemical pathways that play important roles in human physiology and disease. “By combining genuine expertise across biology, chemistry and genetics, Ben has pioneered an approach to drug discovery and development that sits at the heart of Actio’s mission,” said David Goldstein, Ph.D., co-founder and CEO of Actio. “We are thrilled to welcome Ben to our board and look forward to working together to discover and develop new life-changing medicines.” “Years of research has taught us that the best targets are those supported by human genetics – and it is becoming clear that specific kinds of genetic information are especially informative,” said Dr. Cravatt. “Actio’s laser-focused approach targeting genes responsible for rare devastating diseases promises new treatments for those diseases and a powerful framework for rational expansion to more common indications sharing mechanistic biology.” In addition to his role at Scripps, Dr. Cravatt is a co-founder of Activx Biosciences (acquired by Kyorin Pharmaceuticals), Abide Therapeutics (acquired by Lundbeck Pharmaceuticals) and Vividion Therapeutics (acquired by Bayer AG). His honors include a Wolf Prize in Chemistry, a Searle Scholar Award, the Eli Lilly Award in Biological Chemistry, a Cope Scholar Award, the ASBMB Merck Award, the Royal Society of Chemistry Jeremy Knowles Award, and memberships in the National Academy of Sciences, National Academy of Medicine, and American Academy of Arts and Sciences. Dr. Cravatt obtained his undergraduate education at Stanford University, receiving a B.S. in biological sciences and a B.A. in history. He received a Ph.D. from The Scripps Research Institute (TSRI) in 1996 and joined the faculty at TSRI in 1997. About Actio Biosciences Actio Biosciences is leveraging advances in precision medicine to develop new therapeutics that target shared genetics in rare and common diseases—bringing meaningful medicines from one to many. Applying its expertise in genetics, drug discovery and data sciences, Actio seeks to identify programs where both biological and technical risk can be minimized to streamline the drug development process and bring forward exceptionally potent and precisely targeted therapeutics. Founded in October 2021, the San Diego-based company is led by leaders in genetics and drug development and backed by top healthcare investors. For more information, please visit ActioBiosciences.com.

Image Credit: Shutterstock / JHVEPhoto On 14 April 2023, experts from the US Food and Drug Administration’s (FDA) Advisory Committee (AdCom) voted largely in favour of the potential approval of Otsuka’ s and Lundbeck Pharmaceuticals’ Rexulti for the treatment of agitation associated with Alzheimer’s dementia (AAD). Serving as a joint meeting of the Psychopharmacologic Drugs AdCom and the Peripheral and Central Nervous System Drugs AdCom, experts from the committees voted nine against one in support of Rexulti’s supplemental New Drug Application (sNDA) in this condition. At the meeting , Indiana University’s School of Medicine neurology professor Dr. Liana Apostolova said that Rexulti demonstrated statistical significance and resulted in a clinically meaningful therapeutic effect. As a member of the committee, Apostolova voted in favour of Rexulti, adding that she was convinced by the clinical data . The current unmet need, the benefit of treatment with Rexulti, and a reasonable safety profile persuaded Dr. Merit Cudkowicz, Julieanne Dorn professor of neurology at Harvard University’s Medical School, to give a positive vote. In contrast, consumer representative Kim Witczak said that the data does not outweigh the potential risks that come with antipsychotic drugs. Rexulti is an atypical antipsychotic. The FDA will consider the AdCom as it reviews the drug’s sNDA. The agency previously accepted the sNDA in January 2023, setting the PDUFA date to May 10, 2023. The sNDA included data from two positive Phase III studies, based on an April 16 Otsuka update. Both the 331-12-283 and the 331-14-213 studies met their primary endpoints, per the press release. More specifically, in study 331-12-283, a 2mg daily dose of Rexulti was statistically superior to a placebo in the primary endpoint of the mean change in the Cohen-Mansfield Agitation Inventory (CMAI) Total Score from baseline to week 12. If accepted, Rexulti would be the first FDA-approved treatment for AAD. Also known as brexpiprazole, the FDA first approved Rexulti as a treatment for adults with schizophrenia and as an add-on treatment for adults with major depressive disorder in July 2015. However, the treatment’s label features a black box warning that includes increased mortality in elderly patients with dementia-related psychosis treated with antipsychotic drugs. The drug also comes with an increased risk of suicidal thoughts and behaviours in patients aged 24 and younger. There is an urgent need for new treatments of AAD, said Dr. Zahinoor Ismail, professor of psychiatry, neurology, epidemiology, and pathology at the University of Calgary’s Hotchkiss Brain Institute and the O’Brien Institute for Public Health, who presented at the meeting. Overall, there is a need for FDA-approved treatments that reduce AAD symptoms without sedating the patients, said Ismail. He added that there is also a need for FDA-approved products that communicate efficacy and safety on their labels.