Regulations

Kite flies high with second CAR-T approval

Gilead’s Kite has won US FDA approval for Tecartus (brexucabtagene autoleucel), a CAR-T cell therapy for the treatment of mantle cell lymphoma (MCL). Tecartus becomes the third chimeric antigen receptor (CAR) T-cell therapy to be granted approval by the US Food and Drug Administration after Novartis’ Kymriah (tisagenlecleucel) and Kite’s Yescarta (axicabtagene ciloleucel), both of which were approved in 2017. Tecartus is designed to target CD19, a protein found in high amounts in cancerous B cells, and the regulatory decision…

WHO drafting regulatory convergence white paper on cell and gene therapies

Regulators in high-income countries have a responsibility to help health authorities in low- and medium-income countries develop regulatory frameworks for cell and gene therapy products to ensure these treatments are available to all, asserts the US FDA’s biologics center’s director Peter Marks. The World Health Organization plans to soon issue a white paper proposing a harmonized regulatory framework for cell and gene therapy products, according to Peter Marks, director of the US Food and Drug Administration’s Center for Biologics Evaluation…

US FDA says platforms can speed COVID-19 jab development

COVID-19 focused drug and vaccine firms need to keep a close eye on the US FDA according to lawyers who predict manufacturing and trial rules will continue to change. The US Food and Drug Administration (FDA) issued its latest guidance for firms developing vaccines against SARS-CoV-2 – the virus that causes COVID-19 – last week. The document sets out what trial data vaccine firms will need to supply. It also makes clear developers will have to seek approval the traditional…

Manufacturing pace key for COVID-19 vaccines hoping to jump to ‘Warp Speed’

Ease of manufacturing scale up may determine which candidates advance furthest in the US government’s partnership with industry to speed COVID-19 vaccines to patients. Senior government officials also discussed approval standards, how a vaccine would be distributed, and how much the government and Americans could pay in a 16 June press call. The ability for a COVID-19 vaccine to be manufactured quickly and at scale will be a key factor in determining which potential candidates advance through Operation Warp Speed,…

US FDA warns Takeda about Hikari plant

The US FDA has warned Japanese drug firm Takeda about its plant in Hikari, Japan after an inspection conducted last November. According to a letter from the regulator, inspectors who visited the site identified a number of problems with the facility’s quality control unit. The agency wrote, “Our inspection found that your Quality Unit (QU) did not take appropriate steps prior to resumption of aseptic manufacturing after a shutdown that included multiple significant activities that compromised cleanroom control.” It also…

Spark on changing cell and gene regulatory expectations

Regulators are increasingly looking to industry to turn the science of cell and gene therapies into reproducible platform technologies, says Spark Therapeutics’ Diane Blumenthal. The advancements in the cell and gene therapy space were applauded throughout Xconomy’s virtual Xcelerating Philadelphia event last night as industry experts spoke about the increasing number of such therapies moving through the clinic and the hurdles that industry is rapidly knocking over. But challenges continue to exist, and in a panel focused on regulatory pathways,…

Continuous manufacturing back in second Act

House tries again to promote advanced technology for the return of pharmaceutical manufacturing to the US in post-COVID-19 era. The new HEROES Act legislation revives a proposal to establish national centers of excellence for continuous pharmaceutical manufacturing that had earlier made it into the US House of Representatives’ version of the CARES Act but not the final enacted CARES pandemic relief law. The HEROES bill (HR 6800) authorizes $100 million for the centers of excellence, available until expended. That’s up from the $80…

FDA demands more manufacturing info to review ide-cel, says Bluebird

The US FDA has rejected Bluebird Bio’s Biologics license application (BLA) for ide-cel and asked for more details of how the candidate blood cancer therapy is made. Bluebird announced receipt of a “refuse to file letter” (RTF) in an SEC filing on Wednesday. The firm said the US Food and Drug Administration (FDA) asked for supplemental information on validation and control processes used in lentiviral vector and drug product manufacturing processes for Idecabtagene vicleucel (ide-cel), an investigational chimeric antigen receptor…

BioMarin prepares to launch Roctavian for hemophilia in an uncertain environment

The company lowered revenue guidance due to COVID-19, but announced a new brand name, Roctavian, for its hemophilia A gene therapy, which it said remains on track for FDA approval later this year. BioMarin Pharmaceutical is still on track to launch the first gene therapy for hemophilia A in the US later this year, but acknowledged during its first quarter sales and earnings call on 29 April that the commercial dynamics are uncertain because of the COVID-19 outbreak. Management unveiled a…

UK tweaks GMP during pandemic and backs vaccine development

The MHRA relaxed its interpretation of GMP which means UK Biopharma firms have a reduced testing burden during the Covid-19 pandemic. The United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA) announced the move last week, explaining the aim was to let drug firms focus quality system capacity on ensuring supply continuity. Under the plan, firms importing ingredients and finished products will only be required to test to confirm their identity. For other quality attributes importers are permitted to use…