Cell & Gene Therapies
The incoming EU Health Technology Assessment Regulation will see historical data move “more center stage” for advanced therapies, because directly comparing highly individualized therapies is often unfeasible, an advanced therapies expert says.
The US FDA seeks to better understand and predict insertional mutagenesis in gene therapies like bluebird bio's Skysona, which use integrating viral vectors, but a workshop on integration site analysis highlighted the approach's complexity and limitations.
The European Commission’s proposed pharma legislation overhaul could expand the definition of a gene therapy medicinal product, posing challenges both from a regulatory and public perception perspective.
The company said its financial viability is in jeopardy, along with patient access to Lyfgenia, if US FDA Commissioner Robert Califf does not reverse the denial of a rare pediatric disease priority review voucher.
New US FDA draft guidance attempts to address sponsor confusion about the different types of regulatory meetings under PDUFA, as well as frequently asked questions on nonclinical testing, CMC, pharm/tox and clinical studies.
The move towards greater regulatory collaboration is a positive step for gene therapy developers, Astellas’ gene therapy strategy lead Richard Wilson says – adding, however, that pharma still needed to understand the Asian markets better.
Cell and gene therapy manufacturers based in Europe should speak to local regulators to understand how to demonstrate compliance with EU-level good manufacturing practice guidelines, as each country will apply the rules differently, an expert explains.
Lyfgenia and Zynteglo comprise complex mixtures of transduced cells that represent different active ingredients, the company said in administrative appeals to the FDA. Two lawmakers also raised bipartisan concerns about the agency’s decision on the voucher request for the sickle cell disease treatment.
The UK government has introduced draft legislation that will regulate the manufacturing of innovative medicines such as cell and gene therapies at the point of care. The country’s medicines regulator, the MHRA, says the framework is the “first of its kind in the world.”
Regulators from the US, EU, Japan and UK discuss the merits of regulatory convergence for cell and gene therapies, but say that practical challenges often limit their ability to align on pharmaceutical regulatory processes.
Meeting the regulatory gold standard for drug candidates in ultra-rare diseases can be impractical, a regulatory expert says, but greater collaboration and shared insights from regulatory reviews could help find a viable path forward.
Decentralized manufacturing methods for cell and gene therapies will be critical for improving patient access to treatments, but sponsors must prepare to demonstrate “comparability” with centralized manufacturing.
Hemgenix has now secured reimbursement in several European markets, with more talks ongoing. While the path to reimbursement has not always been easy, innovative access deals have helped to smooth the way.
US and EU regulators have invited two companies to work with them on a pilot scheme for assessing drugs for rare genetic diseases, a move that the European Medicines Agency’s Emer Cooke said would help improve “global alignment.”
Long-term follow-up requirements have taken a conservative approach, but could be ripe for re-examination and global harmonization, Kite Pharma executive director says, while former FDA gene/cell therapy office head Wilson Bryan wants the classwide REMS eliminated.
The move is positive for manufacturers and follows the agency’s decision to drop another contentious proposal that would have ‘stacked’ drug discounts when calculating Medicaid best prices.
An FDA question to a patient concerned about the negative impacts of the CAR-T boxed label warning during a recent listening session indicates the agency may be thinking about improving its dissemination of the information.
Japan plans to offer wider support to foreign firms and ventures with innovative candidates to start clinical trials in the country, as part of key measures from current prime minister Fumio Kishida.
The program for collaborative gene therapy reviews with the European Medicines Agency is starting with simple applications, but after launch could be expanded to earlier discussions of CMC and pharm/tox data requirements, the FDA’s Nicole Verdun said.
Regulators in Europe and the US have demonstrated a commitment to providing guidance that will make decentralized and point-of-care manufacturing a reality, the CSO of GermFree, a company that provides mobile and modular cleanrooms for manufacturing advanced therapies, tells the Pink Sheet.