Regulations

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…

Sanofi overcomes CDMO issues with sutimlimab approval

The US FDA approval of Enjaymo (sutimlimab) comes 13 months after Sanofi was hit by a complete response letter citing deficiencies at a third-party manufacturer. The US Food and Drug Administration (FDA) approved Enjaymo Friday, a treatment for patients with rare blood disorder cold agglutinin disease (CAD). The drug, which will be made available in the US at a wholesale acquisition cost of $1,800 per vial, is a humanized monoclonal antibody designed to selectively target and inhibit C1s in the…

FDA 483 shows 7 observations at Catalent fill/finish plant in Belgium

The publication of a US FDA Form 483 reveals failures to implement sustainable corrective action and preventive action, and inadequate maintenance at a Catalent fill/finish facility. Earlier this month, we broke the news that contract development and manufacturing organization (CDMO) Catalent received a Form 483 at its European syringe filling facility in Brussels, Belgium. While the issues observed allegedly caused disruption in the supply of Novo Nordisk’s weight loss drug Wegovy (semaglutide), specific details were sparse. But this week, the…

Catalent hit with FDA 483 at Belgian fill and finish facility

A syringe filling facility in Brussels run by CDMO Catalent has received a US FDA Form 483 and has allegedly created supply issues for Novo Nordisk’s Wegovy. The 265,000 square-foot site described as Catalent’s flagship European syringe filling facility produces over 175 million units annually for the contract development and manufacturing organization’s (CDMO’s) customers. But a recent inspection by the US Food and Drug Administration (FDA) discovered concerns at the site and resulted in a Form 483. “We are treating…

FDA halts US factory inspections amid omicron concerns

The US FDA has temporarily paused its inspectional activities to ensure the safety of its staff and the firms it regulates due to the spread of the omicron variant. The Omicron variant, which was first discovered in November 2021, has already caused global disruption, despite reports from the UK suggesting the risk of hospitalization is about one-third of the Delta variant. With case numbers surging, the US Food and Drug Administration (FDA) decided to implement specific temporary changes to its…