BUSINESS INSIGHTS, PHARMA TECH
EyePoint Pharmaceuticals | February 28, 2023
On February 27, 2023, EyePoint Pharmaceuticals, Inc., a leading pharmaceutical firm committed to the development and commercialization of therapeutics to help improve the lives of patients with severe eye disorders and Rallybio Corporation, a clinical-stage biotechnology company dedicated to identifying and accelerating the development of life-transforming therapeutics for patients with severe and rare diseases, announced a research collaboration to explore the sustained delivery of Rallybio's complement component 5 (C5) inhibitor for the treatment of geographic atrophy, an advanced form of age-related macular degeneration that can cause irreversible vision loss. The research will use EyePoint's proprietary Durasert® technology, which is designed for sustained intraocular drug delivery.
Approximately one million individuals in the United States suffer from geographic atrophy, which is characterized by atrophic lesions in the central region of the macula. Geographic atrophy can cause irreversible vision loss and can lead to legal blindness. Currently, geographic atrophy treatments are limited in choice and duration of action, leaving a significant unmet need for safe and effective treatment options.
Under the terms of the research partnership, EyePoint and Rallybio will assess and explore the viability of utilizing Rallybio's C5 inhibitor in EyePoint's Durasert technology, with the intention to increase the collaboration upon mutual agreement following the evaluation.
EyePoint Pharmaceuticals' M.D., President and Chief Operating Officer Jay Duker commented, "We are excited to begin this research collaboration to explore the combination of Rallybio's C5 inhibitor with our bioerodible Durasert sustained release drug delivery technology to develop a potential long-acting treatment for geographic atrophy," He further emphasized, "Geographic atrophy associated with dry macular degeneration is a devastating eye disease, and the inhibition of complement is a proven treatment pathway. We hope to leverage our Durasert technology in this collaboration to create a potential best-in-class, long-acting intravitreal insert, which we believe could provide a more desirable option for patients given that the existing approved therapy is injected every one to two months."
(Source – Business Wire)
About EyePoint Pharmaceuticals
EyePoint Pharmaceuticals is a leading pharmaceutical company that develops and commercializes therapeutic products to help patients suffering from serious eye disorders. Headquartered in Watertown, Massachusetts, it is committed to using its innovative drug delivery platform to improve patients' quality of life. The company's pipeline utilizes its proprietary Durasert technology, which enables sustained intraocular drug delivery. Its current pipeline includes EYP-1901, a potential six-month intravitreal anti-VEGF treatment aimed at treating wet age-related macular degeneration. Durasert technology has been successfully used across four FDA-approved products, including YUTIQ®, which treats chronic non-infectious uveitis affecting the posterior segment of the eye.
BUSINESS INSIGHTS, PHARMACY MARKET
Xeris Pharmaceuticals, Inc. | January 31, 2023
On January 30, 2023, Xeris Biopharma Holdings, Inc., a leading biopharmaceutical company, announced that the Food and Drug Administration (FDA) granted orphan-drug exclusivity (ODE) to its subsidiary Xeris Pharmaceuticals, Inc. for Recorlev® (levoketoconazole) a cortisol synthesis inhibitor for the treatment of endogenous Cushing's syndrome in adult patients for whom surgery is not viable or has not been effective.
levoketoconazole (Recorlev) being the first FDA-approved therapeutic for the treatment of endogenous hypercortisolemia, Xeris Pharmaceutical is permitted to have seven years of orphan-drug market exclusivity beginning from December 30, 2021. The FDA's Orphan Drug Designation program intends to accelerate the development of medications that treat a disease that affects 200,000 or fewer people in the United States each year. This regulatory exclusivity with the patent exclusivity under Xeris' U.S. patents for Recorlev is valid until at least March 2040.
To serve and assist this community, Xeris is dedicated to ensuring that everyone who needs access to their medications receives them. Xeris has developed Xeris CareConnection™ to provide patients and caregivers with a comprehensive program that includes financial support, one-on-one assistance, and educational materials throughout the treatment journey. Additionally, Xeris CareConnection assists healthcare providers and their teams with education on access and reimbursement.
About Xeris Pharmaceuticals, Inc.
Founded in 2005, Xeris is a growth-oriented biopharmaceutical firm dedicated to improving patients' lives via the development and commercialization of novel products in a range of therapies. Its unique technology makes medicines easier to use for patients & caregivers. The company has three commercially available products; Gvoke®, a ready-to-use liquid glucagon for treating severe hypoglycemia (very low blood sugar); Keveyis®, the first and only FDA-approved therapy for primary periodic paralysis; and Recorlev® for the treatment of endogenous hypercortisolemia. In addition, it has an increasingly broad pipeline of research and partnered projects utilizing its unique formulation technology platforms, XeriSol™ and XeriJect™, bringing forward innovative products for the firm and its partners.
BioMarin Pharmaceutical Inc. | March 08, 2023
On March 7, 2023, BioMarin Pharmaceutical Inc., a global biotech company focused on improving lives via genetic discovery, announced that the US FDA has accepted its supplemental New Drug Application (sNDA) for VOXZOGO® (vosoritide) for injection to expand the treatment of achondroplasia in children under the age of 5.
Achondroplasia is a common form of disproportionate short stature. The FDA has set an action date of October 21, 2023, for the sNDA. The sNDA is supported by data from a Phase 2 double-blind, randomized, placebo-controlled clinical trial, which showed similar safety and efficacy profiles in children under five years of age compared to those aged five years and older.
The European Medicines Agency (EMA) also validated BioMarin's application for VOXZOGO in January to treat children under the age of 2. If approved, VOXZOGO could be prescribed for over 1,000 additional children from birth for achondroplasia.
VOXZOGO is the first EMA and FDA-approved treatment for achondroplasia with open bone growth plates. The treatment uses a new class of therapy, C-type natriuretic peptide (CNP) analog, which promotes bone growth by acting as a positive regulator of the signaling pathway downstream of the fibroblast growth factor receptor 3 gene (FGFR3).
The drug is approved in the EU, Brazil, and Australia for children aged two years and above with epiphyses (bone growth plates) and in Japan for children from birth who have achondroplasia with open growth plates. BioMarin has enrolled 250 children with achondroplasia in seven clinical studies in eight countries to evaluate the safety and efficacy of VOXZOGO.
About BioMarin Pharmaceutical Inc.
BioMarin is a leading biotechnology firm established in 1997 with a mission to transform people's lives by using genetic discovery. The company creates targeted therapies that address the underlying cause of genetic disorders to improve the quality of life for individuals with rare genetic diseases. It has developed eight first or best-in-class treatments and has a range of product candidates to address various genetic disorders using the same science-based approach. As BioMarin continues to create new and innovative solutions, it has the potential to impact the lives of even more people. The company is committed to prioritizing patients and their needs in its approach to drug development.