Pfizer Gets CDSCO Panel Nod To study Osivelotor in sickle cell disease

New Delhi: The Subject Expert Committee (SEC) functional under the Central Drug Standard Control Organisation (CDSCO) has granted approval to drug major Pfizer to conduct the phase 2/3 clinical trial of Osivelotor (also known as PF-07940367 or GBT021601), 25mg and 100mg tablets in participants with sickle cell disease.

However, this approval is subject to the condition that the recruitment should be done from multiple centers from states of Chhattisgarh, Madhya Pradesh and North Eastern States. In addition, the expert panel suggested that safety profile shall be submitted for review before recruitment of adolescent population.

This came after the firm presented Phase 2/3 Clinical trial Protocol No C5351004 Amendment 6 dated 08 Feb 2024. The purpose of this study is to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of GBT021601.

This is a three-part, multicenter, Phase 2/3 study of orally administered GBT021601 in participants with sickle cell disease (SCD). Part A will evaluate the safety, tolerability, and efficacy of GBT021601 in adult participants with SCD to determine an optimal dose. Part B will evaluate the efficacy of GBT021601 versus placebo in adult and pediatric participants with SCD for 48 weeks. Part C will evaluate the pharmacokinetics (PK) and safety of single and multiple doses (MD) of open-label single arm GBT021601 administered to pediatric participants.

Sickle cell disease affects the hemoglobin within red blood cells. A genetic mutation causes abnormal hemoglobin to clump together, causing the red blood cells to turn sickle shaped. These sickle-shaped cells cause blockages in blood flow, which can lead to anemia, pain, infections and severe complications.

Osivelotor is an oral, small molecule, next-generation sickle hemoglobin (HbS) polymerization inhibitor, increases hemoglobin-oxygen (Hb-O2) affinity and stabilizes Hb in the oxy-hemoglobin (oxyHb) state, thereby inhibiting polymerization of HbS in RBCs.

Osivelotor is a tyrosine kinase inhibitor used in the treatment of certain types of non-small cell lung carcinoma.

At the recent SEC meeting for Hematology held on 5th March 2024, the expert panel reviewed the Phase 2/3 clinical trial protocol C5351004 Amendment 6 dated 08 Feb 2024 presented by the drug major Pfizer.

After detailed deliberation, the committee recommended the grant of permission to conduct the clinical trial as presented by the firm with conditions that:

1. The recruitment should be done from multiple centers from states of Chhattisgarh, Madhya Pradesh and North Eastern States.

2. Safety profile shall be submitted for review before recruitment of adolescent population.

Also Read: Need More Justification: CDSCO Panel rejects J&J protocol amendment proposal for Teclistamab study