Amgen Limited has this morning launched Nplate - the first treatment specifically developed for adults with the rare blood disorder immune thrombocytopenic purpura and the first peptibody therapeutic - across the UK.

Chronic ITP is a potentially life-threatening disease characterised by the removal of platelets in the blood by the spleen, which prevent and stop bleeding, affecting around 3,600 adults in the country. While the disease can spontaneously disappear, there is currently no licensed therapy on the market that can cure it.

The availability of Nplate (romiplostim) offers doctors a new strategy for treating and managing the disease, as current therapies – such as immunoglobulins and immunosuppressants – are focused on reducing the rate of platelet destruction while Amgen’s product is the first designed to boost platelet production.

“Nplate is a welcome option for this group of seriously affected adults,” said Dr Drew Provan, Consultant Haematologist at Bart’s & the London School of Medicine. Trial results have been “very encouraging”, he added, as there is a “durable increase in platelet counts and a response is seen within approximately one week”, which, he points out, is “an important step forward in the treatment of ITP”.

Long-lasting benefit
The drug won a regulatory green light in Europe earlier this year on the back of findings from a Phase III trial showing that it raised and sustained platelet counts in 83% of patients whether they had their spleens removed or not. Moreover, an extension study revealed that Nplate continued to effectively increase and sustain platelet counts after three years of therapy, showing that its effects are long-lasting.

Dr Charles Brigden, Medical Director, Amgen UK and Ireland, noted that Nplate is the result of more than 15 years’ research “and represents an important milestone for patients, and for biotechnology, as the first approved peptibody therapeutic”, an engineered protein with attributes of both peptides and antibodies developed by scientists at the company. “It is precisely the sort of targeted, innovative medicine for an unmet need that the UK Government is trying to incentivise through the creation of schemes such as the Office for Life Sciences’ Innovation Pass”, he added.

The drug competes for market share with GlaxoSmithKline’s Promacta (eltrombopag) in the USA, but its rival is yet to receive regulatory approval on this side of the Atlantic, where it is proposed that it will be sold under the trade name Revolade.