Regulations

Rolontis review: Spectrum confident manufacturing deficiencies resolved

Spectrum Pharmaceuticals has resubmitted the BLA for eflapegrastim following the remediation of manufacturing deficiencies at a drug substance facility in Korea. In August 2021, Spectrum received a complete response letter (CRL) from the US Food and Drug Administration (FDA) for its long-acting granulocyte-colony stimulating factor (G-CSF) agent Rolontis (eflapegrastim). The letter, which put a hold on Spectrum’s Biologics License Application (BLA) for eflapegrastim, identified manufacturing challenges. But during his firm’s fourth quarter 2021 financial call last week, CEO Tom Riga…

15 year follow-up for gene editing outlined in FDA draft guide

The US FDA has set a 15 year follow-up period for therapeutic gene editing and edited products in new draft guidelines. The guidance – available here – covers therapeutic use of gene editing techniques like ZFN, and CRISPR, as well as gene therapy products made using those techniques. The aim, according to the FDA, is “to assist in the translation of these products from the bench to clinical trials, this guidance includes recommendations for how to assess the safety and…

J&J taking ‘disciplined’ approach to CAR-T rollout

“CAR-T cell manufacturing is not a finished product,” says J&J EVP Mathai Mammen as he speaks about his firm’s rollout of recently approved myeloma therapy Carvykti. Last month, Johnson & Johnson received the regulatory thumbs up for its chimeric antigen receptor T-cell (CAR-T) therapy Carvykti ( ciltacabtagene autoleucel; ciltacel), developed with China-focused partner Legend Biotech. The therapy is the second BCMA CAR-T approval – Bristol-Myer Squibb’s Abecma (ide-cel) received FDA approval in March 2021 – and the first cell therapy success…

Canada approves plant-based COVID-19 vax

Health Canada has granted authorization for Covifenz, a plant-based COVID-19 vaccine developed by Medicago and GlaxoSmithKline (GSK). Covifenz is made up of plant-based virus-like particles (VLP) and aims to prevent COVID-19 caused by severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) in individuals between the ages of 18 to 64 years old. Medicago uses its Proficia technology to synthesize the virus’ code once a virus has been genetically sequenced. In doing so, the firm claims its genetic instructions can then be…

FDA approves J&J’s myeloma cell therapy

The US FDA has approved a CAR-T therapy developed by Johnson & Johnson and its partner Legend Biotech to treat white blood cell cancer. J&J division Janssen Biotech entered the cell and gene therapy space in 2017, teaming up with Legend Biotech for the development of Carvykti ( ciltacabtagene autoleucel; ciltacel), a chimeric antigen receptor T-cell (CAR-T) therapy that specifically targets the B-cell maturation antigen (BCMA) and is known as LCAR-B38M in China. Now, the US Food and Drug Administration (FDA) has…

EUAs for rare disease advanced therapies? Not likely, says FDA

The FDA has dismissed using special emergency authorization powers for rare disease advanced therapeutics but does hope to significantly speed up its feedback to sponsors. The US Food and Drug Administration’s (FDA) Emergency Use Authorization (EUA) authority allows the use of unapproved medical products to be used in an emergency to diagnose, treat, or prevent serious or life-threatening diseases or conditions caused by chemical, biological, radiological, and nuclear (CBRN) threats when certain criteria are met. Most recently, EUA powers have…

Robert Califf named as FDA commissioner

On Tuesday the Senate voted to confirm Robert Califf as the next commissioner of the US Food and Drug Administration (FDA). The US FDA has not had a permanent leader since January 2021 and in November last year President Joe Biden nominated former FDA commissioner Robert Califf to lead the organization for a second time. Now, in a tight vote resulting in 50 in favor of Califf and 46 against, he returns to the position he served in from 2016…

Lonza cites RealHope to improve DP services

Lonza’s drug product services division is participating in the RealHope project to establish challenges that contribute to protein instability during the handling process. The Real-World Handing of Protein Drugs – Exploration, Evaluation and Education (RealHOPE) project aims to destabilize protein-based therapeutics by measuring real-life events during drug handling to significantly improve the safety and effectiveness of the drug development process. According to Swiss contract development manufacturing organization (CDMO) Lonza, assessing protein stability is required to ensure process optimization and the…

WuXi Bio: COVID-delayed inspections blamed for addition to US Unverified List

The addition of two WuXi Biologics sites to the US Government’s Unverified List will have no impact on its business, the CDMO says. On Monday, the US Commerce Department added 33 Chinese companies to its Unverified List (UVL) including two subsidiaries of contract development and manufacturing organization (CDMO) WuXi Biologics based in Shanghai and Wuxi. As supply chain consultants BDP put it: “The UVL lists names of companies that the Bureau of Industry Security (BIS) could not verify as bona…

Cell and gene sector needs regulatory clarity says Andelyn

Cell and gene therapy developers are still uncertain of regulator expectations according to Andelyn Biosciences, which says industry would benefit from more balanced oversight. Various agencies have invested a lot of effort in the development of regulatory frameworks for cell and gene therapy development and manufacture. Recent examples include the US FDA’s draft guidance on trialing multiple versions of a cell therapy and the EMA’s document on quality expectations for products containing genetically modified cells. While these efforts are a…