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.
BUSINESS INSIGHTS, PHARMACY MARKET
PRNewswire | March 23, 2023
Hovione and Laxxon Medical announced a strategic collaboration to advance the use of 3D screen printing technologies for the pharmaceutical industry. This partnership combines Laxxon´s expertise in 3D screen printing, based on their SPID®-Technology, with Hovione's product & process development, engineering and manufacturing expertise in pharmaceutical applications. Under the terms of the agreement, Hovione will establish the Laxxon Medical 3D screen printing technology at Hovione's cGMP production sites first in Portugal and later in the USA.
The 3D technology displays numerous advantages including the potential to produce unique or customized dosage forms with characteristics that cannot be achieved with conventional dosage forms, the ability to create tablets of any shape and size, the option to easily adjust the number of active substances and individual components in the composition of the tablet and even to set the dosage individually for each patient for personalized medicines.
"Laxxon is thrilled to establish a long-term partnership with Hovione." said Klaus Kuehne, COO of Laxxon Medical. "This partnership will serve as a great accelerator in establishing 3D screen printing within the pharmaceutical industry thus accelerating our ability to serve our customers." "Hovione has a global reputation for their pharmaceutical innovation services, expertise and market access. This agreement marks a significant milestone for Laxxon in terms of our own market strategy and development efforts." said Helmut Kerschbaumer, Laxxon's CEO. "We are looking forward to collaborating with Hovione to promote innovation through 3D screen printing."
"We find the Laxxon Medical 3D printing technology to be very innovative and enabling. It not only allows for the customization and production of drug products with complex shapes and structures as well as unique API release characteristics but it also has the potential to reduce the time and cost of drug development" says Dr. Jean-Luc Herbeaux, Hovione´s CEO. He adds "Hovione has a track record of turning emerging pharmaceutical production technologies into reliable and scalable offerings at both developmental and commercial scales. We are excited at the prospect of supporting Laxxon in industrializing its 3D printing technology and make it accessible to a greater number of pharmaceutical and medical device companies."
With this partnership the platform will be available from early development phases to routine commercial manufacturing.
Hovione is an international company with over 60 years of experience in pharmaceutical development and manufacturing operations. As a Contract Development and Manufacturing Organization (CDMO) Hovione has a fully integrated offering of services for drug substance, drug product intermediate and drug product. The company has four FDA inspected sites in the USA, Portugal, Ireland and China and development laboratories in Lisbon, Portugal and New Jersey, USA. Hovione provides pharmaceutical customers services for the development and compliant manufacture of innovative drugs, including highly potent compounds, and customized product solutions across the entire drug life cycle. In the inhalation area, Hovione offers a complete range of services, from API, formulation development and devices. Hovione´s culture is based on innovation, quality and delivery. Hovione was the first Chemical/ Pharmaceutical Company to become a Certified B Corp, is a member of Rx-360, EFCG and participates actively in industry quality improvement initiatives to lead new global industry standards.
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.