BioMarin gets go-ahead to start gene therapy clinical trials
BMN 307 represents a potential third PKU treatment option from BioMarin and its second gene therapy clinical program.
BioMarin Pharmaceutical, a pioneer in treatments for the rare disease Phenylketonuria (PKU) and in gene therapy clinical research, has announced that both the FDA and the Medicines and Healthcare Products Regulatory Agency (MHRA) have granted the company Investigational New Drug (IND) status and approved its Clinical Trial Application (CTA), respectively, for its investigational gene therapy candidate BMN 307.
BMN 307 is an AAV5-phenylalanine hydroxylase (PAH) gene therapy designed to normalize blood phenylalanine (Phe) concentration levels in patients with PKU. BMN 307 will be evaluated to determine whether a single dose of treatment can restore natural Phe metabolism, normalize plasma Phe levels, and enable a normal diet in patients with PKU.
The company expects to start dosing patients in PHEARLESS, a Phase I/II study, in the first quarter of 2020 with product made at commercial scale from its award-winning gene therapy manufacturing facility. The company is actively preparing regulatory submissions to open additional clinical sites in other countries. BMN 307 represents a potential third PKU treatment option from BioMarin and its second gene therapy clinical program. Both the FDA and European Medicines Agency (EMA) have granted BMN 307 Orphan Status.
BMN 307 follows BioMarin's first investigational gene therapy clinical program: valoctocogene roxaparvovec to treat severe hemophilia A, currently in Phase III. Based on data from an interim analysis of the ongoing Phase III study, the EMA validated a Marketing Authorization Application in the EU for valoctocogene roxaparvovec, and the review process has begun. The company also has submitted a Biologics License Application to the FDA and anticipates the review to begin in February 2020.
"With BMN 307, we are joining together our expertise in PKU biology and the knowledge we have gained from developing the only two approved therapies for PKU with our understanding of gene therapy clinical development and manufacturing from our valoctocogene roxaparvovec experience," said Hank Fuchs, President, Worldwide Research and Development at BioMarin. "BioMarin has stood with the PKU community for over 15 years and remains dedicated to continuing to increase the body of medical knowledge in this devastating disease."
Related News
-
News Novartis signs US$3.2 bn subcutaneous delivery deal with Alteogen
Swiss pharma giant secures access to Korean biotech's ALT-B4 hyaluronidase platform in milestone-heavy agreement, marking Alteogen's fourth major licensing deal of 2026 as competition intensifies in the subcutaneous drug delivery space.
-
News Women in Pharma: The philosophy of pharmaceutical leadership
In this latest instalment of our Women in Pharma series, we sit down with Clarissa Sowemimo-Coker, Chief Strategy Officer at Ambrose Healthcare and CPHI Middle East Advisory Board Member.
-
News The PSCI Supplier Partnership - Collaboration for Responsible Supply Chains
About the Pharmaceutical Supply Chain Initiative The Pharmaceutical Supply Chain Initiative (PSCI) is the leading, member-led association for pharmaceutical companies working collaboratively to build responsible supply chains. Founded in 2006 and es... -
News AstraZeneca shares plunge 8.9% after reports of BMS talks
Britain's largest drugmaker lost more than GBP£17 billion in market value following reports of preliminary merger discussions with Bristol Myers Squibb that could create a pharmaceutical giant worth nearly US$400 billion, though investors rem... -
News GSK announces £400 million investment in new Cambridge R&D centre
Pharmaceutical giant to establish flagship research facility on Cambridge Biomedical Campus as part of strategic pipeline acceleration
-
News The CPHI Sustainability Summit - Where Conversation Becomes Collaboration
The Report Was Never the Destination - “The future of pharmaceutical sustainability won’t be shaped by individual organisations working in isolation. It will be shaped by partnerships, shared expertise and the willingness to work diffe... -
News Trump's generic drug tariffs set to reshape global pharmaceutical supply chains
The US president has announced plans to impose tariffs of up to 200% on imported generic medicines from August 2028, giving manufacturers a two-year window to establish domestic production facilities. The move threatens to upend a global supply chain t... -
News Building Safer, More Responsible Pharmaceutical Supply Chains: PSCI Global Audit Insights
The Pharmaceutical Supply Chain Initiative (PSCI) has released its seventh annual Audit Findings Analysis Report, offering unprecedented insights into supplier practices across the global pharmaceutical and healthcare industry.With a record-breaking 24...