Huge Opportunities Remain for Premium-Priced Tyrosine Kinase Inhibitors in Pulmonary Arterial Hypertension


The Pulmonary Arterial Hypertension (PAH) space, which is currently burdened by significant unmet need, could benefit substantially from a class of drugs known as Tyrosine Kinase Inhibitors (TKIs), which may aid development of a disease-modifying drug, according to an analyst with research and consulting firm GlobalData.

Currently, there are 12 marketed therapies for the treatment of PAH from companies including Actelion, GlaxoSmithKline, United Therapeutics, Bayer, Pfizer, and Gilead Sciences.

Lakshmi Dharmarajan, Ph.D., GlobalData’s Senior Analyst covering Cardiovascular & Metabolic Disorders, says that manufacturing a drug using TKIs could present a significant opportunity for pharmaceutical companies, with the prospect of commanding a high premium over currently existing treatments, which already range in cost from $50,000-$150,000 per year.

Dharmarajan explains: “Previous attempts to develop TKI drugs have met with failure due to the increasing number of concerns regarding the cardiotoxicity of this drug class. For example, Novartis’ cancer blockbuster, imatinib, displayed promising outcomes as a PAH therapy up to Phase III of clinical trials, but severe adverse events caused it to be discontinued.

“Despite these issues, the future prospects of this drug class in PAH treatment continue to be positive. Of course, further research into this class of drugs could be beneficial, since the mechanism of action of TKIs for PAH has already been proven.”

The analyst adds that while all of the current drugs for PAH treatment have the potential to reduce disease progression, none of them are capable of reversing the progression of the disease.

Dharmarajan continues: “TKIs have successfully demonstrated that they can halt cell proliferation, which is a significantly more promising approach than what is currently offered by the late-stage pipeline drugs.

“Indeed, GlobalData believes that focusing on the development of TKI treatments for PAH has the potential not only to produce improved therapies, but also to eventually yield a cure for the disease.”

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