Market News

Global Hemoglobinopathy Treatment Drugs Market: Key Developments

On October 18, 2021, Esteve, a Spain-based international pharmaceutical company, announced U.S. Food and Drug Administration (FDA) approved Seglentis (celecoxib and tramadol hydrochloride), a proprietary product developed by Esteve’s R&D team. It is an innovative first-in-class product comprised of a co-crystal form of celecoxib (an anti-inflammatory) and tramadol (an analgesic) for the treatment of acute pain in adults. This is ESTEVE's first proprietary research product to enter the U.S. market. Seglentis is the trade name for tablets that contain a co-crystal composed of celecoxib and tramadol hydrochloride. It is a new analgesic designed for acute pain management in a multimodal treatment approach targeting four complementary pain relief mechanisms. It offers a new treatment option for acute pain management aligned with the multimodal analgesia now considered standard of care.

On March 22, 2021, Pacira BioSciences, Inc., a U.S. based pharmaceutical company, announced the U.S. Food and Drug Administration (FDA) has approved the submission of its supplemental new drug application (sNDA) seeking expansion of the EXPAREL label to include use in patients 6 years of age and older for single-dose infiltration to produce postsurgical analgesia. With this approval, EXPAREL is the first and only FDA approved long-acting local analgesic for the pediatric population as young as age six. The sNDA was based on the positive data from the Phase 3 PLAY study of EXPAREL infiltration in pediatric patients undergoing surgeries. Overall findings were consistent with the pharmacokinetic and safety profiles for adult patients with no safety concerns identified at a dose of 4 mg/kg. The PLAY study enrolled 98 patients to evaluate safety and the pharmacokinetics of EXPAREL for two patient groups: patients aged 12 to less than 17 years and patients aged 6 to less than 12 years. Per U.S. FDA guidance, the primary objectives of the PLAY study were to evaluate the pharmacokinetics and safety of EXPAREL.

On July 1, 2023, India launched national sickle cell anaemia elimination mission aiming to screen more than seven crore young tribals so that the debilitating disease can be detected early and managed with therapies. The mission, announced in the Union budget earlier in 2023, focus on awareness creation and universal screening of people in the 0-40 years age group in tribal areas in 278 districts in 17 states. The government plans to eliminate the disease by 2047. This mission includes distribution of sickle cell genetic status card to beneficiaries that would save lives of children and families affected by disease. Government have also established integrated centers for hemophilia and hemoglobinopathies in 22 tribal districts for treatment and diagnosis of patients.

On June 21, 2022, Precision BioSciences, Inc., a U.S. based biotechnology company, announced it has entered into an exclusive worldwide in vivo gene editing research and development collaboration and license agreement with Novartis AG, Switzerland based global healthcare company. As part of the Agreement, Precision BioSciences, Inc will develop a custom ARCUS nuclease that will be designed to insert, in vivo, a therapeutic transgene at a safe harbor location in the genome as a potential one-time transformative treatment option for diseases including certain hemoglobinopathies such as sickle cell disease and beta thalassemia. While, Novartis AG taking responsibility for all subsequent research, development, manufacturing, and commercialization activities. Precision will receive an upfront payment of US$ 75 million and is eligible to receive up to an aggregate amount of approximately US$ 1.4 billion in additional payments for future milestones.