BioMarin Pharmaceutical Inc.(NASDAQ: BMRN), a company focused on innovative therapies to treat rare diseases, provided highlights to the investment community on its key milestones for 2019 at the 37th Annual J.P. Morgan Healthcare Conference in San Francisco.
“With multiple development and regulatory milestones expected in 2019, BioMarin maintains strong pipeline momentum. We are looking forward to advancing multiple product candidates in parallel through the development process,” said Jean-Jacques Bienaimé, BioMarin Chairman and CEO. “Our focus on the unmet needs in rare genetic diseases and demonstrating meaningful clinical benefits provides the foundation to move rapidly through product development and commercialization.”
During the company’s presentation at the conference, Bienaimé provided information on the company’s development program for valoctocogene roxaparvovec gene therapy for severe hemophilia A. BioMarin has completed enrollment of the initial cohort of patients in its Phase 3 program intended to support a BLA submission through the accelerated approval pathway. A decision to submit a BLA through an accelerated approval pathway is tracking for the second half of 2019. If the company submits a BLA using the accelerated approval pathway, it will disclose additional information on the timing of its plans regarding the BLA submission. The complete Phase 3 study is targeting enrollment of 130 patients by mid-year 2019.
Valoctocogene roxaparvovec has Orphan Drug designation from the FDA and the European Medicines Agency (EMA). Valoctocogene roxaparvovec has also been accepted for Priority Medicines (PRIME) scheme from the EMA. Additionally, the FDA has granted valoctocogene roxaparvovec Breakthrough Therapy designation.
BioMarin also provided an update on its clinical program for vosoritide, an analog of C-type Natriuretic Peptide (CNP), in children with achondroplasia, the most common form of disproportionate short stature in humans.
BioMarin expects top line results from the fully enrolled Phase 3 study of vosoritide in children by year end 2019. The Phase 2 study in infants and young children up to age 5 with achondroplasia study enrollment is on track, and in this early part of the study, vosoritide is generally well-tolerated. See “About Vosoritide Phase 3 Study” and “About Vosoritide Phase 2 Infant and Young Children Study” below for more information on the studies.
Vosoritide has been granted orphan drug designation in both the U.S. and Europe.
In addition, Bienaimé noted during the presentation that the company is anticipating an opinion from the Committee for Medicinal Products for Human Use (CHMP), the scientific committee of the European Medicines Agency (EMA) on Palynziq® (pegvaliase) Injection for the treatment of patients 16 and older with PKU in 1Q 2019. If the CHMP provides a positive opinion in 1Q 2019, then in 2Q 2019, it is possible that the European Commission (EC) could provide marketing authorization in the European Union. In addition, the company provided 2019 global revenue guidance for Palynziq of between $70 and $100 million.
Palynziq, a PEGylated recombinant phenylalanine ammonia lyase enzyme approved in the United States in May 2018, is the first approved enzyme substitution therapy to target the underlying cause of phenylketonuria (PKU) by helping the body to break down Phe. PKU is a rare genetic disease that manifests at birth and results in a variety of cumulative toxic effects on the brain. Palynziq is BioMarin’s second approved treatment for this serious condition. In March 2018, the European Medicines Agency accepted BioMarin’s submission of a Marketing Authorization Application for Palynziq.
Gene Therapy Manufacturing
BioMarin has constructed one of the largest gene therapy manufacturing facilities of its kind, which is located in Novato, California. Good Manufacturing Practices (GMP) production of valoctocogene roxaparvovec has commenced supporting clinical development activities and will support any anticipated commercial demand. This facility is capable of supporting approximately 4,000 doses per year, and the production process was developed in accordance with International Conference on Harmonisation guidance for Pharmaceuticals for Human Use to facilitate worldwide registration with health authorities. Clinical supplies for valoctocogene roxaparvovec for Hemophilia A and BMN 307, a pre-clinical gene therapy for PKU, are produced in this facility.
No comments:
Post a Comment
Note: Only a member of this blog may post a comment.