The US Food and Drug Administration (FDA) on Tuesday approved a new gene therapy from Spark Therapeutics for the treatment of children and adult patients with an inherited form of vision loss that may result in blindness. The approval comes two months and a week after an advisory committee of outside experts unanimously voted in favor of approving the treatment for the condition which affects about 6,000 worldwide. The price of the new gene therapy, which Leerink...

This article provides an overview of biomedical applications of gene editing technology, addresses ethical and regulatory challenges associated with its implementation for therapeutic development, and proposes approaches for overcoming these challenges. Introduction Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) technology is a genetic "editing" tool aimed at altering DNA sequences and modifying gene function. It offers promising opportunities for ...

The US Food and Drug Administration (FDA) and the Centers for Medicare and Medicaid Services (CMS) on Thursday announced joint approval and coverage decisions for Foundation Medicine's FoundationOne CDx next generation sequencing (NGS) based test. The test was reviewed under FDA and CMS' parallel review program, and is only the second test to be approved and cleared under the program since its inception in 2011, the first being Exact Sciences' Cologuard colorectal cance...

At a Senate Health, Education, Labor and Pensions (HELP) hearing in Washington, D.C. on Tuesday, experts explained that the current regulatory framework for gene editing in the US is appropriate and cautioned that an overly strict approach could drive research to other countries. Specifically, the hearing focused on CRISPR/Cas9, which is a gene editing tool that scientists can use to quickly and accurately edit DNA in a variety of applications. While there are curr...

The European Medicines Agency (EMA) on Friday said that its Committee for Medicinal Products for Human Use (CHMP) is recommending six new drugs, a biosimilar and three generic medicines for EU approval. The six medicines the agency is recommending are: Dr. Falk Pharma's Jorveza (budesonide) to treat eosinophilic esophagitis, a rare inflammatory condition of the esophagus. Jorveza was granted an accelerated assessment and orphan designation; Merck Sharp & Dohme's Pr...

The US Food and Drug Administration (FDA) on Tuesday issued a draft guidance detailing the agency's new program for breakthrough medical devices created by the 21st Century Cures Act . The new breakthrough devices program supersedes and combines several of the agency's existing programs to speed access to new devices. "Earlier and more frequent interaction between the FDA and manufacturers … should allow manufacturers to make the best use of their resources to bring st...

The European Medicines Agency (EMA) and European Commission on Friday published a joint action plan to further the development of new cell and gene therapies, as well as other advanced therapy medicinal products (ATMPs). The plan, which features the release of several new and revised guidelines comes as on the international level, a regular forum for dialogue has been established to share experiences on advanced therapies between EMA, the US Food and Drug Administr...

Philadelphia-based Spark Therapeutics took its gene therapy before the US Food and Drug Administration’s (FDA) Cellular, Tissue and Gene Therapies Advisory Committee on Thursday and the panel voted 16 to 0 in favor of approving the subretinal injection, representing a major step forward for adeno-associated virus gene therapies. The therapy, known as Luxturna (voretigene neparvovec), has been granted priority review by FDA (the agency has until 12 January 2018 t...

The US Food and Drug Administration (FDA) on Tuesday issued its final guidance on classifying combination products as drugs, biologics or medical devices. The guidance finalizes and combines two 2011 draft guidances, Classification of Products as Drugs and Devices & Additional Product Classification Issues and Interpretation of the Term 'Chemical Action' in the Definition of Device Under Section 201(h) of the Federal Food, Drug and Cosmetic Act , and details how F...

The US Food and Drug Administration (FDA) is increasingly granting faster reviews via expedited regulatory pathways, but a new article published in the British Medical Journal found a higher association with these expedited pathways and the likelihood of safety-related labeling changes than with non-expedited pathways. In their analysis of 15 years of data, authors Sana Mostaghim, Joshua Gagne and Aaron Kesselheim of the Program on Regulation, Therapeutics, And Law...

The US Food and Drug Administration (FDA) on Wednesday approved the first gene therapy, Novartis' Kymriah (tisagenlecleucel) for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL). Dubbed an "historic action" by the agency, the approval comes a little more than a month after FDA's Oncologic Drugs Advisory Committee voted unanimously , 10 to 0, in favor of the benefit-risk profile for the first of this new kind of cancer th...

The US Food and Drug Administration's (FDA) Commissioner Scott Gottlieb said Monday the agency will advance a new framework to better regulate stem cell therapies this fall. "This comprehensive policy will establish clearer lines around when these regenerative medicine products have sufficient complexity to fall under the agency's current authority, and then define an efficient process for how these products should be evaluated for safety and effectiveness," Gottli...