New treatment for Cushing’s disease gets green light

Isturisa (osilodrostat) oral tablets have been approved in the US for adults with Cushing’s disease who either cannot undergo pituitary gland surgery or have undergone the surgery but still have the disease.

Cushing’s disease is a rare disease in which the adrenal glands make too much of the cortisol hormone.

Made by Novartis, Isturisa is the first FDA-approved drug to directly address this cortisol overproduction by blocking the enzyme known as 11-beta-hydroxylase and preventing cortisol synthesis.

Isturisa’s safety and effectiveness for treating Cushing’s disease among adults was evaluated in a study of 137 adult patients (about three-quarters women) with a mean age of 41 years.

The majority of patients either had undergone pituitary surgery that did not cure Cushing’s disease or were not surgical candidates.

In the 24-week, single-arm, open-label period, all patients received a starting dose of 2 mg of Isturisa twice a day that could be increased every two weeks up to 30 mg twice a day. At the end of this 24-week period, about half of patients had cortisol levels within normal limits.

After this point, 71 patients who did not need further dose increases and tolerated the drug for the last 12 weeks entered an eight-week, double-blind, randomized withdrawal study where they either received Isturisa or a placebo (inactive treatment).

At the end of this withdrawal period, 86% of patients receiving Isturisa maintained cortisol levels within normal limits compared to 30% of patients taking the placebo.

Made by Novartis, Isturisa received Orphan Drug Designation.

A message from the Editor:

Thank you for reading this story on our news site - please take a moment to read this important message:

As you know, our aim is to bring you, the reader, an editorially led news site but journalism costs money and we rely on advertising and digital revenues to help to support them.

With the Covid-19 lockdown having a major impact on our industry as a whole, the advertising revenues we normally receive, which helps us cover the cost of our journalists and this website, have been drastically affected.

As such we need your help. If you can support our news sites with a small donation of even £1, your generosity will help us weather the storm and continue in our quest to deliver quality journalism.

In the meantime may I wish you the very best.

- Advertisement -

Joint investment sees UK gov secure pipeline of coronavirus vaccine candidate

The UK government and biotech Valneva have jointly invested in a Scottish facility to bolster vaccine manufacturing capacity. The multi-million-pound investment will allow the government...

Redx out-licenses porcupine inhibitor to AstraZeneca

UK drug discovery and development company, Redx Pharma, has signed a significant out-licensing agreement for its porcupine inhibitor, RXC006, with AstraZeneca. AstraZeneca will take RXC006...

New Chief Medical Officer for Valneva

Valneva, a specialty vaccine company based in France, has appointed Juan Carlos Jaramillo, as Chief Medical Officer and member of the Management Board. Starting 1st...

Majority of top pharma firms report YoY growth in 2019 – data

In 2019, 17 of the top twenty major pharmaceutical players reported year-on-year revenue growth, according to GlobalData. “One winner was Takeda, which saw more than...

Teva & Alvotech join forces to commercialise biosimilars in US

Israel’s Teva Pharmaceutical and Icelandic biopharma Alvotech have entered into an exclusive strategic partnership for the commercialisation in the US of five biosimilar product...

Related news

Blenrep becomes first anti-BCMA approved globally

GlaxoSmithKline (GSK) has secured US approval for Blenrep (belantamab mafodotin-blmf) as a monotherapy treatment for adult patients with relapsed or refractory multiple myeloma. This indication...

Joint investment sees UK gov secure pipeline of coronavirus vaccine candidate

The UK government and biotech Valneva have jointly invested in a Scottish facility to bolster vaccine manufacturing capacity. The multi-million-pound investment will allow the government...

Redx out-licenses porcupine inhibitor to AstraZeneca

UK drug discovery and development company, Redx Pharma, has signed a significant out-licensing agreement for its porcupine inhibitor, RXC006, with AstraZeneca. AstraZeneca will take RXC006...

New Chief Medical Officer for Valneva

Valneva, a specialty vaccine company based in France, has appointed Juan Carlos Jaramillo, as Chief Medical Officer and member of the Management Board. Starting 1st...