Oxbryta®

Update: 19 December 2024

• Withdrawal and recall was announced by the company of voxelotor from all approved markets and suspension of ongoing clinical trials and expanded access programmes.
• The EMA initiated a review of the drug (July 2024) due to an imbalance of risks observed in clinical trials. The CHMP recommended suspending marketing authorisation.
• 8 deaths occurred in the treatment arm of the clinical trial (total 236 patients participated). The company is further reviewing the available data.
• The U.S. Food and Drug Administration is conducting a safety review of post-marketing clinical data for the drug, and real-world registry studies.

Sources: https://www.pfizer.com/news/press-release/press-release-detail/pfizer-voluntarily-withdraws-all-lots-sickle-cell-disease
https://www.reuters.com/business/healthcare-pharmaceuticals/pfizer-withdraws-sickle-cell-disease-treatment-all-markets-2024-09-25/#:~:text=In%20a%20study%20of%20236,deaths%20in%20the%20placebo%20arm.
https://www.ema.europa.eu/en/documents/referral/oxbryta-article-20-procedure-review-started_en.pdf
https://www.ema.europa.eu/en/news/ema-recommends-suspension-sickle-cell-disease-medicine-oxbryta
https://www.reuters.com/business/healthcare-pharmaceuticals/pfizers-oxbryta-exit-may-hasten-trials-rival-experimental-sickle-cell-drugs-2024-09-26/

Update: 30 September 2024

No update available.

Update: 30 June 2024

Data presented at the 29^th EHA Annual Congress (13 – 16 June 2024) in Madrid (Spain) showed that in the open-label extension HOPE trial, long-term use of voxelotor (for 4+ years) by 199 patients,  long-term exposure to voxelotor may reduce RBC transfusions and VOCs.

This is consistent with previous analyses that showed voxelotor was well tolerated and effective at improving haemoglobin and haemolysis parameters.

Source: Long-Term Effect of Voxelotor on Red Blood Cell Transfusions and Vaso-Occlusive Crises in Patients with Sickle Cell Disease: Results From an Open-Label Extension of The Phase 3 Hope Trial

Update: 31 March 2024

• Data from the HOPE-KIDS 1 phase 2a clinical trial (NCT02850406) has shown positive results in reducing sickling of RBCs. The study aims to understand how Oxbryta works and the findings support the hypothesis that RBC deformability is reduced as Oxbryta increased the binding of oxygen to haemoglobin.
• Oxbryta is approved in the U.S. to treat SCD in adults and children as young as 4. It’s approved for patients 12 and older in the European Union, the U.K., and some countries in the Middle East.
• NICE (UK) has re-opened its review into Oxbryta and requested additional evidence on its cost-effectiveness. Final decision expected in June 2024.

Sources: https://sicklecellanemianews.com/news/oxbryta-prevents-red-blood-cell-sickling-scd-children-study/
https://www.thepharmaletter.com/article/slow-progress-on-new-treatments-for-sickle-cell-disease-in-the-uk
https://pink.citeline.com/PS149868/Pfizer-Ponders-Next-Move-After-English-HTA-Rejects-Oxbryta-Again-Despite-Appeal
https://www.nice.org.uk/guidance/indevelopment/gid-ta10505

Update: 20 December 2023

NICE has announced that it will reconsider the product, after its previous rejection due to insufficient evidence of long-term or wide population effectiveness. The reconsideration comes after an appeal lodged by the Sickle Cell Society, UK Haemoglobinopathy Group and the company.

New data

Data presented at the 65^th ASH Annual Congress (9 –12 December 2023) in San Diego (USA) showed that:

• • In the open-label extension HOPE trial, long-term use of voxelotor (for 4+ years) was well tolerated and effective at achieiving low rates of anaemia, haemolysis and VOCs (178 patients)
  • A post-marketing patient registry in the USA of patients who receive voxelotor in a real-world setting (PROSPECT; NCT04930445) has been established. At the data cutoff (May 30, 2023), 85 patients were enrolled at 5 sites. The registry will follow patients for up to 5 years to enable long-term, robust data collection of the real-world.

Sources: https://www.nursingtimes.net/news/policies-and-guidance/transformative-sickle-cell-disease-treatment-closer-to-approval-10-12-2023/
https://ash.confex.com/ash/2023/webprogram/Paper188616.html
https://ash.confex.com/ash/2023/webprogram/Paper188746.html

Update: 30 September 2023

No update available.

Update: 30 June 2023

No update available.

Update: 31 March 2023

Preliminary draft guidance from England’s HTA body NICE states the product is not an acceptable use of National Health Service resources. Previously the UK regulatory body MHRA grants marketing authorisation approval in 2022.

Oxbryta® (voxelotor) is intended for the treatment of haemolytic anaemia due to Sickle Cell Disease (SCD) in eligible adult and paediatric patients 12 years of age and older as monotherapy or in combination with hydroxycarbamide (hydroxyurea).

NICE will proceed to a public consultation on the preliminary recommendation, before the final recommendation later in the year.

Sources: https://pink.pharmaintelligence.informa.com/PS147563/Blow-For-Pfizer-As-UKsNICE-Rejects-Oxbryta-For-Sickle-Cell-Disease
https://www.pfizer.co.uk/news/media/pfizers-response-nice-decision-oxbryta