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The company is discontinuing all active trials of the sickle cell disease treatment, voxelotor, globally. Credit: Sebastian Kaulitzki / Shutterstock

Pfizer has announced the voluntary withdrawal of all lots of OXBRYTA (voxelotor) approved for treating sickle cell disease (SCD) from worldwide markets.

The company is also discontinuing all active voxelotor clinical trials and expanded access programmes globally.

The decision comes after the report of comprehensive clinical data that suggested that the risks of OXBRYTA may outweigh the benefits for the approved patient population.

The findings also indicated an imbalance in vaso-occlusive issues and fatal events which called for additional assessment.

The company has informed regulatory authorities of these findings, and its decision to halt the distribution of OXBRYTA and cease ongoing clinical studies.

An oral, once-daily treatment, OXBRYTA was designed to increase the oxygen affinity of haemoglobin to hinder sickle haemoglobin polymerisation.

The US Food and Drug Administration (FDA) initially granted accelerated approval for OXBRYTA tablets in 2019 for treating SCD in adults and children aged 12 years and above.

Teva Pharmaceuticals has shared new safety data from the Phase III SOLARIS trial and a Phase I study of extended-release subcutaneous olanzapine, TEV-‘749, showing that none of the patients in the two studies experienced post-injection delirium/sedation syndrome (PDSS).

Olanzapine is an atypical antipsychotic and was first approved by the US Food and Drug Administration (FDA) in 2009. Eli Lilly markets it as Zyprexa and a longer-acting version as Zyprexa Relprevv. Generics of the therapy are also available, however, its use as a long-acting treatment has been limited as it is linked with a risk of developing PDSS. The condition causes the sudden onset of delirium or sedation within hours of receiving treatment. Zyprexa was a breakthrough product for Lilly, generating peak sales of over $3bn in 2001. Pharmaceutical Technology‘s parent company GlobalData expects Teva’s olanzapine extended-release product to reach annual sales of $145m by 2034 in the US.

Sanofi will gain exclusivity rights to Ventyx Biosciences’ CNS-penetrant candidate VTX3232 as part of a $27m deal.

Under the terms of the agreement, Sanofi will buy 70,601 shares of Ventyx’s Series A non-voting convertible preferred stock at $3.82 per share of common stock. As per the 23 September press release, each share of preferred stock is “initially convertible” into 100 shares of common stock. The purchase, which is expected to be sufficient to fund operations into H2 2026, is set to finalise within the day.

In return, Sanofi will gain an exclusive right to first negotiation for the candidate.The announcement caused Ventyx’s stock prices to increase by 14.8% when the market opened on 23 September. The company’s stock price remains considerably lower than a year ago, having dropped by 92.2% since 25 September 2023. 

The National Health Service (NHS) England has announced the routine commissioning of Daiichi Sankyo‘s quizartinib for acute myeloid leukaemia (AML) patients with the FLT3-ITD mutation.

The decision, facilitated by the Cancer Drugs Fund, will enable immediate access to the life-extending daily tablet, which has been shown to improve remission and survival rates significantly.

The treatment, now available for prescription, is intended to be used in conjunction with chemotherapy for newly-diagnosed adult patients and may be continued as a maintenance treatment for up to three years to minimise the relapse risk.

For patients showing significant improvement following quizartinib treatment, a stem cell transplant will be offered.

For those ineligible for stem cell transplants, the tablet could still offer a substantial benefit by prolonging life.

The UK’s Medicines and Healthcare products Regulatory Agency previously confirmed the asset’s safety and efficacy, citing a median overall survival of 31.9 months with quizartinib against just 15.1 months in the placebo arm.

The US Food and Drug Administration (FDA) has declined to approve the new drug application (NDA) submitted by Vanda Pharmaceuticals for tradipitant to treat gastroparesis symptoms. 

The decision was communicated through a complete response letter (CRL). In December 2023, the FDA accepted a new drug application (NDA) seeking approval for the asset.

The application for tradipitant was supported by evidence from two placebo-controlled studies, bolstered by exposure response data from a large open-label study and the real-world experiences of patients in an expanded access programme.

However, the CRL dismisses the evidence and suggests further studies with a design and duration that Vanda believes to be inconsistent with expert advice and the scientific understanding of gastroparesis.

In a media release, Vanda Pharmaceuticals stated: “The FDA’s action was delayed by more than 185 days and fails to satisfy the requirements specified by the Food Drug and Cosmetic Act (FDCA).