Luspatercept

 

‘BELIEVE’ Study in Adults with TDT β-thalassaemia

 

Update: 27 September 2019

No update available

 

Update: 30 July 2019

  • The FDA has accepted the Biologics License Application (BLA) for luspatercept. The FDA has granted Priority Review to this BLA for the evaluation of the beta-thalassemia indication and set a Prescription Drug User Fee Act (PDUFA), or target action date of December 4, 2019. This is the date by which the FDA must review the application.

Sources:

https://ir.celgene.com/press-releases/press-release-details/2019/Celgene-Corporation-and-Acceleron-Pharma-Announce-US-FDA-Accepts-Luspatercept-Biologics-License-Application-in-Myelodysplastic-Syndromes-and-Beta-Thalassemia/default.aspx

 

Update: 30 May 2019

  • Application for Marketing Authorization to the European Medicines Agency (EMA) for luspatercept for the treatment of adult patients with beta-thalassemia-associated anemia who require RBC transfusions has been submitted.

 

  • Submission to the FDA Biologics License Application (BLA) for luspatercept. A BLA  is submitted after an investigational new drug has been approved and constitutes a request for permission to introduce, or deliver for introduction, a biologic product into interstate commerce.

Sources:

https://ir.celgene.com/press-releases/press-release-details/2019/Celgene-Corporation-and-Acceleron-Pharma-Announce-Submission-of-Luspatercept-Marketing-Authorization-Application-to-the-European-Medicines-Agency-EMA-for-MDS-and-Beta-Thalassemia/default.aspx

https://ir.celgene.com/press-releases/press-release-details/2019/Celgene-Corporation-and-Acceleron-Pharma-Announce-Submission-of-Luspatercept-Biologics-License-Application-to-US-FDA/default.aspx

 

Update: 29 March 2019

Regulatory updates have been announced indicating that Luspatercept Biologics License Application (BLA) will be submitted in April 2019.

A BLA  is submitted after an investigational new drug has been approved and constitutes a request for permission to introduce, or deliver for introduction, a biologic product into interstate commerce.

Sources:

https://ir.celgene.com/press-releases/press-release-details/2019/Celgene-Corporation-Announces-Key-Regulatory-Updates-for-REVLIMID-in-Lymphoma-and-Luspatercept-in-MDS-and-Beta-Thalassemia/default.aspx

 

Update: 02 October 2018

Luspatercept is an erythroid maturation agent (EMA) that is believed to regulate late-stage red blood cell maturation.

Results were announced from the Phase 3, randomized, double-blind, multi-centre ‘BELIEVE’ clinical study for the evaluation of efficacy and safety of luspatercept in adult β-thalassaemia patients.

Main results:

  • Luspatercept achieved a highly statistically significant improvement in the primary endpoint of erythroid response. This was defined as at least a 33 % reduction from baseline in red blood cell (RBC) transfusion burden with a reduction of at least 2 units during the protocol-defined period of 12 consecutive weeks, from week 13 to week 24, compared to placebo. Essentially this means that participants saw more than a 33% reduction in their blood transfusion requirements over a period of 12 consecutive weeks, thus indicating that Luspatercept reduces the need for blood transfusions.
  • Luspatercept also met all key secondary endpoints of demonstrating statistically significant improvements in RBC transfusion burden from baseline of at least a 33% reduction during the period from week 37 to week 48, at least a 50% reduction during the period from week 13 to week 24, at least a 50% reduction during the period from week 37 to week 48, and a mean change in transfusion burden from week 13 to week 24.

Thus, the results show a significant reduction in transfusion burden compared to placebo.

Further results with be announced during the Annual ASH Congress.

Regulatory submissions are planned for the FDA and EMA in the first half of 2019, with market access anticipated in early 2020.

Source: https://ir.celgene.com/press-releases/press-release-details/2018/Celgene-and-Acceleron-Announce-Luspatercept-Achieved-Primary-and-All-Key-Secondary-Endpoints-in-Phase-III-BELIEVE-Study-in-Adults-with-Transfusion-Dependent-Beta-Thalassemia/default.aspx

 

Update: 20 December 2018

Results from a pivotal, phase 3 trial (BELIEVE) evaluating the safety and efficacy of luspatercept for the treatment of adults with transfusion dependent β-thalassaemia were presented at the 60th Annual Meeting of the American Society of Hematology (ASH) in San Diego, held on 01 – 04 December 2018.

  • RBC Transfusion Burden (i.e. number of RBC units required) reduced by more than 33%
  • The mean change was -1.35 RBC units
  • Treatment-emergent adverse events were reported by 29.1% of participants, mainly anaemia (3.1%), increased LIC (2.7%), Hyperuricemia (2.7%) and others.
  • Other side effects include bone pain, backache, and hypertension.

Source: https://ir.celgene.com/press-releases/press-release-details/2018/Celgene-Corporation-and-Acceleron-Pharma-Announce-Results-of-the-Phase-3-BELIEVE-Trial-Evaluating-Luspatercept-in-Adult-Patients-with-Beta-Thalassemia-at-ASH-2018/default.aspx

 

‘BEYOND’ Study in Adults with NTDT β-thalassaemia

 

Update: 30 May 2019

No update available.

 

Update: 02 October 2018

Phase 2 trials are ongoing to ascertain safety and efficacy.

Source: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5796131/#B99-ijms-19-00182

 

Update: 20 December 2018

Announcement of results are scheduled for early 2020.

Source:Email correspondence with company.