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Specialty Pharma Expert Adopts Name of Global Parent to Reflect Breadth of Offer That Brings a Rare and Special Perspective to the Market

Businesswire | April 26, 2023 | Read time : 02:00 min

Specialty Pharma Expert Adopts

Global healthcare and business consulting services provider, Terebellum, has chosen to adopt the name of its global parent, AscellaHealth, to reflect the scale of its operations and breadth of expertise.

The business specialises in providing end-to-end solutions to pharmaceutical manufacturers and other industry stakeholders to streamline product commercialisation, improve medication access, reduce costs and enhance patient outcomes for specialty and rare disease patients.

Based in Dublin, Ireland and Manchester, UK, former Terebellum provides pre-commercialisation and market access support, supply chain logistics, distribution and fulfilment and unique pharmaceutical financial services to UK and European pharmaceutical manufacturers and healthcare organisations.

Adopting the brand name of its parent company, AscellaHealth, will enable the specialty pharma expert to demonstrate its full range of expertise, international insight and distinctiveness of offer. Upon Terebellum’s transition to the AscellaHealth brand identity in its market-facing activities, the company will continue to maintain current business operations, client relationships and dedication to patient-centric care.

Craig Caceci, Managing Director at AscellaHealth EU/UK, said: “It’s our strong belief that to optimise outcomes in specialty healthcare a specialised approach is needed, due to the specific challenges associated with rare conditions.

“The AscellaHealth name provides an opportunity to highlight how we are perfectly positioned to offer uniquely tailored solutions across the globe to ensure streamlined medication access and the best possible treatment journey for rare and orphan disease patients.”

In a recent customer survey carried out by AscellaHealth, 100 percent of respondents agreed that the organisation presents unique value in the market and is uniquely qualified to address their needs.

Craig adds: “Supporting patients, life science manufacturers, payers and providers, we bring expertise and care to every requirement to develop and implement novel approaches that ensure patients with rare diseases can get the treatment they need, when they need it most.

“Our new brand tagline, ‘a rare and special perspective’, captures our dedication to applying knowledge and expertise to improve health outcomes of rare disease and specialty patients, perfectly.”

AscellaHealth, co-founded by Dea Belazi, President & CEO and Bill Oldham, Chairman & CFO in 2013, employs nearly 200 employees worldwide to provide integrated end-to-end solutions that span the entire pharmaceutical lifecycle and beyond, supporting all segments of the specialty pharma industry.

Former Terebellum joins US-based pharmacy distribution and patient-management organisation, Optime Care, in adopting the name of parent AscellaHealth, to form one globally-recognised brand for specialty pharma expertise.

About AscellaHealth LLC

AscellaHealth, a global Healthcare & Specialty Pharmacy solutions organisation, serving patients, life sciences manufacturers, payers, and providers, offers a comprehensive portfolio of uniquely tailored, tech-enabled services supporting complex, chronic conditions or rare diseases that require specialty medications and/or cell and gene therapies. A recipient of numerous industry awards for innovation, and a NASP Strategic Channel Partner of the Year award winner, AscellaHealth’s best-in-class, patient-centric approach is built upon proprietary technology processes for novel programs and services to support the launch of specialty medications and proactively address multiple challenges, optimise clinical health outcomes, and improve quality of life for this patient population. AscellaHealth brings a rare and special perspective to all stakeholders.

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Catalyst Clinical Research Announces the Acquisition of Genpro Research

PRNewswire | July 21, 2023

Catalyst Clinical Research an industry-leading provider of specialized clinical development solutions, announced today it has acquired Genpro Research ("Genpro"), a Massachusetts-based global clinical research organization with staff in the US, India, and Ireland. Genpro is a next generation services and technology partner for the pharmaceutical, biotechnology, and medical devices industry with expertise in biometrics, medical writing, RWE, and AI enabled automation product development. Genpro will continue to be led by Dr. Sachin Marulkar and the existing management team. Commenting on the announcement, Catalyst CEO Nick Dyer said, "We are hugely enthusiastic about the addition of Genpro to our family. We share the opportunity to expand our global footprint, broaden and deepen our services, and to use the AI platform to create value for ourselves and customers in a range of labor-intensive document creation processes. In addition, our approach to prioritizing culture and treating our teams like family are similar and will act as a critical foundation for our combined company as we move forward." Genpro CEO Dr. Sachin Marulkar added, "By joining Catalyst, we will continue to expand our biometrics, scientific reporting and technology offerings, have access to more opportunities globally, and provide our clients with additional products and services. We are excited to utilize the synergies in advancing our AI/ML technology platform to create beneficial new tools and improve the efficiency of clinical research. Catalyst is highly aligned with our purpose, values, and our relationship-driven focus. We look forward to stepping into these opportunities while still acting with the speed and flexibility our customers value and maintaining the collaborative culture that is so important to our employees." This acquisition adds more than 120 team members in India with expertise in biometrics and medical writing further bolstering Catalyst's robust functional service offerings. This further enables biopharma, MedTech and CRO clients (current and future) to access Catalyst's specialized clinical research services, along with broader expertise in global time zones, new solutions, and early access to innovative automation platforms. Melissa Church, Senior Vice President, Strategy at Catalyst, expressed the strategic significance of the acquisition, emphasizing its importance in advancing Catalyst's commitment to providing top-notch specialized clinical development solutions. She stated, "Catalyst is extremely intentional in its acquisition approach, ensuring we choose organizations that are additive to our capabilities and match well with our people-first culture. In Genpro we are gaining a very talented team delivering function specific biometrics and medical writing services which fits squarely in our strategic growth plan supporting both our Catalyst Flex and Catalyst Oncology solutions. This creates more value for our customers, collaborators, partners and investors long term." Highlighting scientific and innovation focus of the deal, Kapil Khambholja, Genpro's CSO and Practice Head of Medical Writing, Real-World Evidence, and Health Economics and Outcomes Research (RWE & HEOR) emphasised both company's shared commitment for advancing therapies beyond milestones to transforming lives. Dr Kapil stated, "This partnership will propel Genpro's automation roadmap forward, aligning strategically with Catalyst's interests. We are also thrilled to see Genpro's scientific initiatives gaining momentum, and we eagerly anticipate collaborating with Catalyst's dedicated team to inspire advancements of breakthrough therapies touching lives." About Catalyst Clinical Research Catalyst is a clinical research organization (CRO) that provides highly customizable solutions to the global biopharmaceutical industry through two established, branded solutions: Catalyst Oncology and Catalyst Flex. The company provides full-service oncology CRO offerings through Catalyst Oncology and multi-therapeutic Client- or Catalyst-managed functional services through Catalyst Flex. With around 1000 staff and offices in the United States, Europe, and Asia-Pacific regions, Catalyst's flexible service model is built from more than a decade of listening to customers, devising customer-centric solutions, and helping customers drive breakthrough clinical studies by leveraging expert teams and innovative technologies. Catalyst is a portfolio company of QHP Capital, a leading healthcare and life sciences investment firm. About Genpro Research Genpro Research is a technology and services partner dedicated to serving the pharmaceutical, biotechnology, and medical devices industries. By providing expertise in biostatistics, data sciences, medical writing & evidence synthesis, the focus has been on thoughtful service execution on clinical studies. And, as an experienced technology provider, Genpro uses Artificial Intelligence, Machine Learning, and Natural Language Processing algorithms for clinical development and post-launch activities, Genpro's central commercial platform called MaiA is used for Evidence Generation and Study Report Automation that brings depth and clarity to enable informed decision making.

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Ginkgo Bioworks Advances Collaboration with Novo Nordisk on Expression Systems for Pharmaceutical Products

PRNewswire | July 13, 2023

Ginkgo Bioworks which is building the leading platform for cell programming and biosecurity, today announced the completion of the pilot phase of their previously announced partnership with Novo Nordisk, a leading global healthcare company, to develop expression systems for pharmaceutical products. With the pilot phase completed and the first milestone achieved, the two companies will now move into the development phase to work towards making a more effective production process. Achievement of the first milestone included extensive modification and testing of a microbial expression system to understand the impact of the modifications on key performance parameters. "Ginkgo's achievement of the first milestone has both excited and inspired us, giving us the confidence to move into the next phase of this ambitious project," said Brian Vandahl, Senior Vice President, Global Research Technologies at Novo Nordisk. "This progress is a strong indicator of the potential we have to engineer biological systems that will expand the chemical space of biological medicines." "We're excited to continue our work with Novo Nordisk to help provide innovative treatment options for patients with diabetes, obesity, and other serious chronic diseases," said Jason Kelly, CEO and co-founder, Ginkgo Bioworks. "The next phase of this project will build on our existing microbial design, engineering, and phenotyping expertise while expanding Ginkgo's capabilities in genome-scale design and engineering. We believe this work will enable us to support even more pharmaceutical products, helping to achieve better outcomes for patients." About Ginkgo Bioworks. Ginkgo Bioworks is the leading horizontal platform for cell programming, providing flexible, end-to-end services that solve challenges for organizations across diverse markets, from food and agriculture to pharmaceuticals to industrial and specialty chemicals. Ginkgo's biosecurity and public health unit, Concentric by Ginkgo, is building global infrastructure for biosecurity to empower governments, communities, and public health leaders to prevent, detect, and respond to a wide variety of biological threats.

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Catalyst Pharmaceuticals Acquires Exclusive North American License For Vamorolone for Duchenne Muscular Dystrophy from Santhera Pharmaceuticals

Globenewswire | July 20, 2023

Catalyst Pharmaceuticals, Inc. announced the completion of its acquisition from Santhera Pharmaceuticals Holdings of an exclusive license for North America for vamorolone, a potential treatment for patients suffering with Duchenne Muscular Dystrophy. The license is for exclusive commercial rights in the U.S., Canada, and Mexico, as well as the right of first negotiation in Europe and Japan should Santhera pursue partnership opportunities. Additionally, Catalyst will hold North American rights for any future approved indications of vamorolone. Vamorolone is a promising best-in-class dissociative anti-inflammatory steroid treatment for Duchenne Muscular Dystrophy ("DMD"). In clinical studies, vamorolone demonstrated efficacy with a significant reduction of steroid-associated side effects and benefits for bone health, growth, and behavior, offering the potential to address an important unmet medical need in DMD patients. Vamorolone has received FDA Orphan Drug and Fast Track designations and has been granted a PDUFA action date of October 26, 2023. "With the addition of vamorolone, we have accomplished another important milestone in the execution of our portfolio expansion strategy," stated Patrick J. McEnany, Chairman and CEO of Catalyst. "The license for vamorolone reflects our strategic focus on opportunities where we can not only apply novel technology to address critical unmet patient needs, but where we can leverage Catalyst's existing integrated capabilities and infrastructure to commercialize the drug efficiently. We believe that vamorolone, if approved, has the potential to deliver significant near and long-term value and to be a very meaningful advancement to the current standard of care paradigm for DMD patients." Mr. McEnany continued, "Vamorolone fortifies our neuromuscular portfolio with an innovative therapy that, in clinical studies, demonstrated an enhanced safety and tolerability profile as compared to prednisone. We plan to include vamorolone for DMD patients in our Catalyst Pathways® specialty pharmacy program to help ensure that all patients have access to the full patient benefits of the program. Our planned strategy to facilitate access to vamorolone underscores our steadfast commitment to improving the lives of patients suffering from rare neurological conditions." Duchenne Muscular Dystrophy, or DMD, the most common form of muscular dystrophy, is a rare, fatal neuromuscular disorder characterized by progressive muscular dysfunction, leading to loss of ambulation, respiratory failure, and death. Corticosteroids are the current standard of care for treating DMD; however, this treatment is associated with significant side effect burdens. The U.S. prevalence for DMD is estimated to be between 11,000 and 13,000 patients. Of patients currently being treated for DMD, approximately 75% receive concomitant steroid treatment. Vamorolone Commercial Operational Plan Catalyst expects to launch vamorolone early in the first quarter of 2024, assuming regulatory approval on the PDUFA date of October 26, 2023. Catalyst anticipates minimal sales and marketing personnel expansion with fewer than 10 additional team members required, resulting from the exceptional synergy within its existing neuromuscular franchise. Catalyst plans to incorporate vamorolone for DMD into its Catalyst Pathways® specialty pharmacy program to ensure that patients have access to full patient benefits. About Vamorolone Vamorolone is an investigational drug candidate with a mode of action based on binding to the same receptor as glucocorticoids but modifying its downstream activity and as such, is considered a dissociative anti-inflammatory steroid drug [2-5]. This mechanism has shown the potential to 'dissociate' efficacy from steroid safety concerns, and therefore vamorolone could emerge as an alternative to existing corticosteroids, the current standard of care in children, adolescent, and adult patients with DMD. In the pivotal VISION-DMD study, vamorolone met the primary endpoint Time to Stand (TTSTAND) velocity versus placebo (p=0.002) at 24 weeks of treatment and showed a good safety and tolerability profile [1]. The most commonly reported adverse events versus placebo from the VISION-DMD study were cushingoid features, vomiting, and vitamin D deficiency. Adverse events were generally of mild to moderate severity. Vamorolone has been granted Orphan Drug status for DMD in the U.S. and Europe and has received Fast Track and Rare Pediatric Disease designations by the U.S. FDA and Promising Innovative Medicine (PIM) status from the UK MHRA for DMD. Vamorolone is an investigational medicine and is currently not approved for use by any health authority. About Duchenne Muscular Dystrophy Duchenne Muscular Dystrophy ("DMD") is a rare inherited X-chromosome-linked disease, which almost exclusively affects males. DMD is characterized by muscle inflammation and damage which are present at birth or shortly thereafter. Inflammation leads to fibrosis of muscle and is clinically manifested by progressive muscle degeneration and weakness. Major milestones in the disease are the loss of ambulation, the loss of self-feeding, the start of assisted ventilation, and the development of cardiomyopathy. DMD reduces life expectancy to before the fourth decade due to respiratory and/or cardiac failure. Corticosteroids are the current standard of care for the treatment of DMD. About Catalyst Pharmaceuticals With exceptional patient focus, Catalyst is committed to developing and commercializing innovative first-in-class medicines that address rare neurological and epileptic diseases. Catalyst's U.S. commercial product portfolio consists of FIRDAPSE® (amifampridine) Tablets 10 mg, approved for the treatment of Lambert-Eaton myasthenic syndrome ("LEMS") for adults and children ages six to seventeen. In January 2023, Catalyst acquired the U.S. commercial rights of FYCOMPA® (perampanel) CIII, a prescription medicine approved in people with epilepsy aged four and older alone or with other medicines to treat partial-onset seizures with or without secondarily generalized seizures, and with other medicines to treat primary generalized tonic-clonic seizures for people with epilepsy aged 12 and older. Further, Canada's national healthcare regulatory agency, Health Canada, has approved the use of FIRDAPSE for the treatment of adult patients in Canada with LEMS.

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Catalyst Clinical Research Announces the Acquisition of Genpro Research

PRNewswire | July 21, 2023

Catalyst Clinical Research an industry-leading provider of specialized clinical development solutions, announced today it has acquired Genpro Research ("Genpro"), a Massachusetts-based global clinical research organization with staff in the US, India, and Ireland. Genpro is a next generation services and technology partner for the pharmaceutical, biotechnology, and medical devices industry with expertise in biometrics, medical writing, RWE, and AI enabled automation product development. Genpro will continue to be led by Dr. Sachin Marulkar and the existing management team. Commenting on the announcement, Catalyst CEO Nick Dyer said, "We are hugely enthusiastic about the addition of Genpro to our family. We share the opportunity to expand our global footprint, broaden and deepen our services, and to use the AI platform to create value for ourselves and customers in a range of labor-intensive document creation processes. In addition, our approach to prioritizing culture and treating our teams like family are similar and will act as a critical foundation for our combined company as we move forward." Genpro CEO Dr. Sachin Marulkar added, "By joining Catalyst, we will continue to expand our biometrics, scientific reporting and technology offerings, have access to more opportunities globally, and provide our clients with additional products and services. We are excited to utilize the synergies in advancing our AI/ML technology platform to create beneficial new tools and improve the efficiency of clinical research. Catalyst is highly aligned with our purpose, values, and our relationship-driven focus. We look forward to stepping into these opportunities while still acting with the speed and flexibility our customers value and maintaining the collaborative culture that is so important to our employees." This acquisition adds more than 120 team members in India with expertise in biometrics and medical writing further bolstering Catalyst's robust functional service offerings. This further enables biopharma, MedTech and CRO clients (current and future) to access Catalyst's specialized clinical research services, along with broader expertise in global time zones, new solutions, and early access to innovative automation platforms. Melissa Church, Senior Vice President, Strategy at Catalyst, expressed the strategic significance of the acquisition, emphasizing its importance in advancing Catalyst's commitment to providing top-notch specialized clinical development solutions. She stated, "Catalyst is extremely intentional in its acquisition approach, ensuring we choose organizations that are additive to our capabilities and match well with our people-first culture. In Genpro we are gaining a very talented team delivering function specific biometrics and medical writing services which fits squarely in our strategic growth plan supporting both our Catalyst Flex and Catalyst Oncology solutions. This creates more value for our customers, collaborators, partners and investors long term." Highlighting scientific and innovation focus of the deal, Kapil Khambholja, Genpro's CSO and Practice Head of Medical Writing, Real-World Evidence, and Health Economics and Outcomes Research (RWE & HEOR) emphasised both company's shared commitment for advancing therapies beyond milestones to transforming lives. Dr Kapil stated, "This partnership will propel Genpro's automation roadmap forward, aligning strategically with Catalyst's interests. We are also thrilled to see Genpro's scientific initiatives gaining momentum, and we eagerly anticipate collaborating with Catalyst's dedicated team to inspire advancements of breakthrough therapies touching lives." About Catalyst Clinical Research Catalyst is a clinical research organization (CRO) that provides highly customizable solutions to the global biopharmaceutical industry through two established, branded solutions: Catalyst Oncology and Catalyst Flex. The company provides full-service oncology CRO offerings through Catalyst Oncology and multi-therapeutic Client- or Catalyst-managed functional services through Catalyst Flex. With around 1000 staff and offices in the United States, Europe, and Asia-Pacific regions, Catalyst's flexible service model is built from more than a decade of listening to customers, devising customer-centric solutions, and helping customers drive breakthrough clinical studies by leveraging expert teams and innovative technologies. Catalyst is a portfolio company of QHP Capital, a leading healthcare and life sciences investment firm. About Genpro Research Genpro Research is a technology and services partner dedicated to serving the pharmaceutical, biotechnology, and medical devices industries. By providing expertise in biostatistics, data sciences, medical writing & evidence synthesis, the focus has been on thoughtful service execution on clinical studies. And, as an experienced technology provider, Genpro uses Artificial Intelligence, Machine Learning, and Natural Language Processing algorithms for clinical development and post-launch activities, Genpro's central commercial platform called MaiA is used for Evidence Generation and Study Report Automation that brings depth and clarity to enable informed decision making.

Read More

Pharmacy Market

Ginkgo Bioworks Advances Collaboration with Novo Nordisk on Expression Systems for Pharmaceutical Products

PRNewswire | July 13, 2023

Ginkgo Bioworks which is building the leading platform for cell programming and biosecurity, today announced the completion of the pilot phase of their previously announced partnership with Novo Nordisk, a leading global healthcare company, to develop expression systems for pharmaceutical products. With the pilot phase completed and the first milestone achieved, the two companies will now move into the development phase to work towards making a more effective production process. Achievement of the first milestone included extensive modification and testing of a microbial expression system to understand the impact of the modifications on key performance parameters. "Ginkgo's achievement of the first milestone has both excited and inspired us, giving us the confidence to move into the next phase of this ambitious project," said Brian Vandahl, Senior Vice President, Global Research Technologies at Novo Nordisk. "This progress is a strong indicator of the potential we have to engineer biological systems that will expand the chemical space of biological medicines." "We're excited to continue our work with Novo Nordisk to help provide innovative treatment options for patients with diabetes, obesity, and other serious chronic diseases," said Jason Kelly, CEO and co-founder, Ginkgo Bioworks. "The next phase of this project will build on our existing microbial design, engineering, and phenotyping expertise while expanding Ginkgo's capabilities in genome-scale design and engineering. We believe this work will enable us to support even more pharmaceutical products, helping to achieve better outcomes for patients." About Ginkgo Bioworks. Ginkgo Bioworks is the leading horizontal platform for cell programming, providing flexible, end-to-end services that solve challenges for organizations across diverse markets, from food and agriculture to pharmaceuticals to industrial and specialty chemicals. Ginkgo's biosecurity and public health unit, Concentric by Ginkgo, is building global infrastructure for biosecurity to empower governments, communities, and public health leaders to prevent, detect, and respond to a wide variety of biological threats.

Read More

Pharmacy Market

Catalyst Pharmaceuticals Acquires Exclusive North American License For Vamorolone for Duchenne Muscular Dystrophy from Santhera Pharmaceuticals

Globenewswire | July 20, 2023

Catalyst Pharmaceuticals, Inc. announced the completion of its acquisition from Santhera Pharmaceuticals Holdings of an exclusive license for North America for vamorolone, a potential treatment for patients suffering with Duchenne Muscular Dystrophy. The license is for exclusive commercial rights in the U.S., Canada, and Mexico, as well as the right of first negotiation in Europe and Japan should Santhera pursue partnership opportunities. Additionally, Catalyst will hold North American rights for any future approved indications of vamorolone. Vamorolone is a promising best-in-class dissociative anti-inflammatory steroid treatment for Duchenne Muscular Dystrophy ("DMD"). In clinical studies, vamorolone demonstrated efficacy with a significant reduction of steroid-associated side effects and benefits for bone health, growth, and behavior, offering the potential to address an important unmet medical need in DMD patients. Vamorolone has received FDA Orphan Drug and Fast Track designations and has been granted a PDUFA action date of October 26, 2023. "With the addition of vamorolone, we have accomplished another important milestone in the execution of our portfolio expansion strategy," stated Patrick J. McEnany, Chairman and CEO of Catalyst. "The license for vamorolone reflects our strategic focus on opportunities where we can not only apply novel technology to address critical unmet patient needs, but where we can leverage Catalyst's existing integrated capabilities and infrastructure to commercialize the drug efficiently. We believe that vamorolone, if approved, has the potential to deliver significant near and long-term value and to be a very meaningful advancement to the current standard of care paradigm for DMD patients." Mr. McEnany continued, "Vamorolone fortifies our neuromuscular portfolio with an innovative therapy that, in clinical studies, demonstrated an enhanced safety and tolerability profile as compared to prednisone. We plan to include vamorolone for DMD patients in our Catalyst Pathways® specialty pharmacy program to help ensure that all patients have access to the full patient benefits of the program. Our planned strategy to facilitate access to vamorolone underscores our steadfast commitment to improving the lives of patients suffering from rare neurological conditions." Duchenne Muscular Dystrophy, or DMD, the most common form of muscular dystrophy, is a rare, fatal neuromuscular disorder characterized by progressive muscular dysfunction, leading to loss of ambulation, respiratory failure, and death. Corticosteroids are the current standard of care for treating DMD; however, this treatment is associated with significant side effect burdens. The U.S. prevalence for DMD is estimated to be between 11,000 and 13,000 patients. Of patients currently being treated for DMD, approximately 75% receive concomitant steroid treatment. Vamorolone Commercial Operational Plan Catalyst expects to launch vamorolone early in the first quarter of 2024, assuming regulatory approval on the PDUFA date of October 26, 2023. Catalyst anticipates minimal sales and marketing personnel expansion with fewer than 10 additional team members required, resulting from the exceptional synergy within its existing neuromuscular franchise. Catalyst plans to incorporate vamorolone for DMD into its Catalyst Pathways® specialty pharmacy program to ensure that patients have access to full patient benefits. About Vamorolone Vamorolone is an investigational drug candidate with a mode of action based on binding to the same receptor as glucocorticoids but modifying its downstream activity and as such, is considered a dissociative anti-inflammatory steroid drug [2-5]. This mechanism has shown the potential to 'dissociate' efficacy from steroid safety concerns, and therefore vamorolone could emerge as an alternative to existing corticosteroids, the current standard of care in children, adolescent, and adult patients with DMD. In the pivotal VISION-DMD study, vamorolone met the primary endpoint Time to Stand (TTSTAND) velocity versus placebo (p=0.002) at 24 weeks of treatment and showed a good safety and tolerability profile [1]. The most commonly reported adverse events versus placebo from the VISION-DMD study were cushingoid features, vomiting, and vitamin D deficiency. Adverse events were generally of mild to moderate severity. Vamorolone has been granted Orphan Drug status for DMD in the U.S. and Europe and has received Fast Track and Rare Pediatric Disease designations by the U.S. FDA and Promising Innovative Medicine (PIM) status from the UK MHRA for DMD. Vamorolone is an investigational medicine and is currently not approved for use by any health authority. About Duchenne Muscular Dystrophy Duchenne Muscular Dystrophy ("DMD") is a rare inherited X-chromosome-linked disease, which almost exclusively affects males. DMD is characterized by muscle inflammation and damage which are present at birth or shortly thereafter. Inflammation leads to fibrosis of muscle and is clinically manifested by progressive muscle degeneration and weakness. Major milestones in the disease are the loss of ambulation, the loss of self-feeding, the start of assisted ventilation, and the development of cardiomyopathy. DMD reduces life expectancy to before the fourth decade due to respiratory and/or cardiac failure. Corticosteroids are the current standard of care for the treatment of DMD. About Catalyst Pharmaceuticals With exceptional patient focus, Catalyst is committed to developing and commercializing innovative first-in-class medicines that address rare neurological and epileptic diseases. Catalyst's U.S. commercial product portfolio consists of FIRDAPSE® (amifampridine) Tablets 10 mg, approved for the treatment of Lambert-Eaton myasthenic syndrome ("LEMS") for adults and children ages six to seventeen. In January 2023, Catalyst acquired the U.S. commercial rights of FYCOMPA® (perampanel) CIII, a prescription medicine approved in people with epilepsy aged four and older alone or with other medicines to treat partial-onset seizures with or without secondarily generalized seizures, and with other medicines to treat primary generalized tonic-clonic seizures for people with epilepsy aged 12 and older. Further, Canada's national healthcare regulatory agency, Health Canada, has approved the use of FIRDAPSE for the treatment of adult patients in Canada with LEMS.

Read More

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