Ipsen | January 24, 2022
Ipsen announced the Health Canada approval of Sohonos, an oral selective retinoic-acid receptor gamma agonist indicated to reduce the formation of heterotopic ossification in adults and children aged 8 years and above for females and 10 years and above for males with fibrodysplasia ossificans progressiva.1 Sohonos is approved for the treatment of patients with FOP for both chronic use, and for flare-ups, in these patient populations. This decision marks the first approval for Sohonos worldwide.
“FOP is a progressive and debilitating condition which has such a profound impact on patients, and their families. Until today, there was no approved medicine, and we are proud to bring this important new medicine to the FOP community.”
Dr. Howard Mayer, Executive Vice President and Head of Research and Development, Ipsen
FOP is characterized by new bone formation outside of the normal skeletal system, like in soft connective tissues, a process known as heterotopic ossification,2 which can be preceded by painful soft tissue swelling or “flare-ups.”2 Flare-up episodes are common and are a substantial contributor to the formation of new bone, however bone can also form in the absence of a flare-up. Once formed, it is irreversible and leads to loss of mobility and shortened life expectancy.2 It is an ultra-rare genetic disorder with an estimated prevalence of 1.36 per million individuals; however, the number of confirmed cases varies by country.3,4.
As part of the ongoing commitment to the FOP and rare disease community, Ipsen plans to file in the US in H1 2022 and is in discussions with other regulatory authorities around the world.
About the MOVE clinical program
This approval is based on data from the ongoing MOVE trial, the first global multi-center Phase III trial in FOP. MOVE is an open-label, single-arm trial, evaluating the efficacy and safety of a chronic/flare-up dosing regimen of palovarotene in decreasing new annualized HO volume in patients with FOP.
Sohonos is an oral, selective RARγ agonist developed as a treatment for people living with the debilitating ultra-rare genetic disorder, FOP. The treatment was acquired by Ipsen through the acquisition of Clementia Pharmaceuticals in April 2019. It is a member of the retinoid class of drugs that is associated with birth defects in humans. Sohonos must not be used by patients who are, or intend to become, pregnant due to the risk of teratogenicity. To minimize fetal exposure, Sohonos is to be administered only if all conditions for pregnancy prevention are met. Sohonos has been shown to cause premature physeal closure in growing children with FOP; periodic monitoring is recommended.
Ipsen is a global, mid-sized biopharmaceutical company focused on transformative medicines in Oncology, Rare Disease and Neuroscience; it also has a well-established Consumer Healthcare business. With total sales of over €2.5bn in FY 2020, Ipsen sells more than 20 medicines in over 115 countries, with a direct commercial presence in more than 30 countries. The Company’s research and development efforts are focused on its innovative and differentiated technological platforms located in the heart of leading biotechnological and life-science hubs: Paris-Saclay, France; Oxford, U.K.; Cambridge, U.S.; Shanghai, China. Ipsen has around 5,700 colleagues worldwide and is listed in Paris and in the U.S. through a Sponsored Level I American Depositary Receipt program.
Veeva Systems | May 21, 2021
A growing number of life sciences companies are adopting Veeva Development Cloud applications from Veeva Systems in an industrywide shift to streamline drug development. quite 650 companies use Veeva Development Cloud applications today, and quite 250 are leveraging multiple Veeva Vault applications across functional areas to enable and connect drug development and manufacturing teams.
Veeva Development Cloud brings together suites of unified applications for clinical, regulatory, quality, and safety on one cloud platform, eliminating organizational siloes. quite 350 companies are accelerating trial execution with Vault Clinical applications, quite 250 are streamlining regulatory processes with Vault RIM, quite 350 are unifying quality management with Vault Quality, and quite 35 are improving pharmacovigilance with Vault Safety.
Vault Connections in Veeva Development Cloud drive cross-functional business processes across the merchandise lifecycle. For instance, Connections enable companies to compile clinical operations and regulatory for faster submissions; quality and regulatory for simplified change control and variation management; and clinical data and operations for real-time visibility into patient enrollment.
"We're dedicated to bringing innovations to plug that help the industry accelerate drug development," said Jim Reilly, vice chairman of Veeva Vault R&D. "Veeva Development Cloud is enabling customers to enhance collaboration across teams and speed the delivery of treatments to patients."
About Veeva Systems
Veeva is that the global leader in cloud software for the life sciences industry. Committed to innovation, product excellence, and customer success, Veeva serves quite 975 customers, starting from the world's largest pharmaceutical companies to emerging biotechs. As a Public Benefit Corporation, Veeva is committed to balancing the interests of all stakeholders, including customers, employees, shareholders, and therefore the industries it serves.
Orsini Specialty Pharmacy | September 28, 2021
Orsini Specialty Pharmacy, the leading independent specialty pharmacy focused on rare diseases and gene therapies, announced that Sanofi has selected it as a limited distribution partner for Nexviazyme® (avalglucosidase alfa-ngpt). Nexviazyme, an enzyme replacement therapy (ERT), is used for treating patients one year of age and older with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency). This progressive and debilitating muscle disorder impairs a person's ability to move and breathe. For more information about Nexviazyme, please see the Full Prescribing Information.
Pompe disease affects an estimated 3,500 people in the United States. It can present as infantile-onset Pompe disease (IOPD), the most severe form of Pompe disease with rapid onset in infancy, and late-onset Pompe disease (LOPD), which progressively damages muscles over time. LOPD symptoms may present at any age. However, due to the broad spectrum of clinical presentations and progressive nature of the disease, it can take seven to nine years before patients receive an accurate diagnosis. As the disease progresses, people with LOPD may require mechanical ventilation to help with breathing or a wheelchair to assist with mobility.
Orsini's dedicated Pompe Disease Care Team's number one priority is providing exceptional care and compassionate support to patients and their families, We are proud to expand our partnership with Sanofi and add Nexviazyme to the growing number of enzyme replacement therapies we dispense to patients with rare diseases.
- Mike Fieri, Orsini's Founder and Chief Executive Officer.
About Orsini Specialty Pharmacy
Providing patients with comprehensive and compassionate care since 1987, Orsini is the leading independent specialty pharmacy focused on rare diseases, gene therapies, and complex conditions. Orsini's high-touch care model centers around experienced, therapy-specific care teams that provide personalized care to patients based on their specific conditions and treatments. The company's comprehensive solutions include medication adherence programs, data analytics, customized manufacturer programs, and nationwide nursing coverage for convenient in-home infusion services. Headquartered in Elk Grove Village, IL, Orsini Specialty Pharmacy holds accreditations with URAC, the Accreditation Commission for Health Care (ACHC), ACHC's Distinction in Rare Diseases and Orphan Drugs, The Joint Commission, and the National Association of Boards of Pharmacy (NABP).