Hengrui gets world-first okay for platelet drug hetrombopag

Jiangsu Hengrui Pharma's oral thrombopoietin (TPO) receptor agonist hetrombopag has been approved in China to treat low platelet counts (thrombocytopenia) caused by chemotherapy for solid tumours.

The green light from the National Medical Products Administration (NMPA) makes the drug the first oral TPO agonist to be approved for chemotherapy-induced thrombocytopenia (CIT) anywhere in the world, according to Hengrui.

Hetrombopag has, meanwhile, also been approved by the NMPA for second-line use in paediatric patients aged six and over with immune thrombocytopenia (ITP), adding to its existing use in adults with the rare autoimmune blood disorder, as well as adolescents and adults with severe aplastic anaemia (SAA).

Thrombocytopenia is a common complication of chemotherapy for solid tumours, raising the risk of bleeding side effects and – in severe cases – requiring dose reductions that can compromise the efficacy of the cancer treatment and place burdens on health systems through the need for platelet transfusions and other supportive care.

Injectable TPO products are already being used to treat CIT in China – namely 3SBio's Tpiao (rhTPO), which has been approved by the NMPA for this indication, and romiplostim biosimilars, which are used off-label – these require multiple clinic visits and add to the burden on patients and hospitals.

"There is an urgent clinical need for treatment options with both efficacy and convenience of medication, so as to facilitate the progress of anti-tumour treatment as planned," said Hengrui in a statement.

"The approval of this new indication makes this product the first […] TPO receptor agonist approved for [CIT] in the world, which will provide strong support for anti-tumour treatment and promote the standardised management […] throughout the whole process."

The approval in CIT, meanwhile, gives Hengrui an indication for hetrombopag free of competition from other drugs in the oral TPO category, namely Novartis' Revolade (eltrombopag) and generics, Fosun Pharma and Sobi's Doptelet (avatrombopag), and Shionogi/Eddingpharm's Mulpleta (lusutrombopag).

Among these, Doptelet and Revolade have been tested in clinical trials for CIT, but generated mixed results, while Mulpleta's developers are focusing on thrombocytopenia associated with chronic liver disease.

Citing EvaluatePharma data, Hengrui said that global sales of oral TPO drugs were approximately $2.2 billion last year.