Globenewswire | May 15, 2023
Viridian Therapeutics, Inc. a biotechnology company focused on discovering and developing potential best-in-class medicines for serious and rare diseases, announced a new partnership with Enable Injections, a company developing and manufacturing the enFuse® innovative wearable drug delivery system for volumes of up to 25mL.
“As we expand our pipeline beyond TED and assess the unmet needs of patients living with other serious and rare diseases, it’s clear that convenient drug delivery is an important issue,” said Scott Myers, President and CEO of Viridian. “We believe Enable’s proprietary technology can contribute meaningfully to our goal of delivering therapeutic advances while reducing patient treatment burden as well.”
“Enable Injections is excited to partner with Viridian to utilize the enFuse® technology to enhance the patient experience,” said Michael D. Hooven, Enable Injections' Chairman and CEO. “The enFuse® on-body delivery system was engineered with the patient in mind—the hidden needle and hands-free delivery allows patients to have increased flexibility, improved convenience, and the ability to administer medication discreetly, whether at home via self-administration or in the clinic.”
This partnership, which applies exclusively to areas outside of Viridian's established TED portfolio, underscores the company's commitment to patient-centric innovation.
About Viridian Therapeutics
Viridian Therapeutics is a biopharmaceutical company focused on engineering and developing potential best-in-class medicines for patients with serious and rare diseases. Viridian’s expertise in antibody discovery and engineering enables it to develop differentiated therapeutic candidates for previously validated drug targets in commercially established disease areas.
Viridian is advancing multiple candidates in the clinic for the treatment of patients with thyroid eye disease (TED). The Company has initiated its first global Phase 3 trial called ‘THRIVE’ to evaluate the safety and efficacy of VRDN-001 in patients with active TED. Viridian is also evaluating VRDN-001 in a Phase 2 proof-of-concept trial in patients with chronic TED. In addition to its program for intravenously administered VRDN-001, the Company is advancing three candidates for its subcutaneous strategy with the goal of providing a more conveniently administered therapy to patients with TED. Viridian is also developing multiple preclinical assets in autoimmune and rare diseases.
BUSINESS INSIGHTS, PHARMA TECH
Prnewswire | April 13, 2023
Flexan LLC announced that it is expanding its services to include custom manufacturing of sterile liquids and custom formulated solutions for a variety of medical device applications. Flexan is partnering with its parent company, ILC Dover, and its KSE Scientific division to expand offerings to its medical device customers. KSE Scientific provides standard and custom formulations and packaging while following the most stringent quality standards. The company is FDA registered for medical devices and 510(k) for sterile water and saline. With over 50,000L of Water for Injection (WFI) production capacity per day and more than 113,000 square feet of manufacturing, controlled storage, and laboratory space, KSE Scientific is ready to solve customers' challenges for sterile liquids.
Recently, both Flexan and KSE Scientific partnered to deliver a custom solution and packaging service to Clyra Medical Technologies.
"Clyra is pleased to find a high-quality partner under one roof in KSE Scientific and Flexan, to support manufacturing for our best-in-class wound management and surgical product 'Bioclynse'. Bioclynse is 510(k) cleared by the FDA, and is an efficacious, safe, long-acting wound irrigation solution that can be used during wound management, during surgery and for post-surgical wounds, with no need to rinse out throughout the entire procedure," said Steve Harrison, CEO of Clyra Medical Technologies. "We look forward to working with KSE Scientific and Flexan to support solution manufacturing and developing customized packaging and components to ensure we meet the market demand and customer requirements for delivering our solutions."
"Flexan serves a broad range customers in the medical device market and we are excited to expand our services to include custom sterile fluids for various clinical applications. In addition to adding sterile fluids, we are also excited to share that we are now offering platinum-cured silicone tubing, tubing assemblies and molded components to better serve ILC's biopharmaceutical customers worldwide," said Tony Gonzalez, President of Flexan.
About ILC Dover
ILC Dover is a world-leader in the innovative design and production of solutions for biopharmaceutical, pharmaceutical, medical device markets as well as a leading supplier for the (aero)space industries. Our customers will attest to our relentless dedication to high value products, advanced technology, and responsive service, as our visionary solutions have improved efficiency while safeguarding people, product, and infrastructure in hazardous conditions through flexible protective solutions since 1947.
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.