Immune Thrombocytopenia (ITP) is diagnosed by low platelet counts in the blood. It also causes impaired platelet production. This put patients at risk for serious bleeding as well as spontaneous bruising.
10 years ago Nplate® (romiplostim) was approved as a treatment for adults with Immune Thrombocytopenia. However, the drug was not approved for pediatric patients with the disease, leaving those who were unresponsive to other forms of therapy out of luck. Treatment options for children with the disease included immunoglobulins, corticosteroids, or a splenectomy. But now, they have another option.
Nplate has just been approved by the FDA for use in children one year and older who have had the condition for at least 6 months and have had an inadequate response to other treatments. The drug’s supplemental Biologics License Application (sBLA) was sent in by Amgen, and approval was announced on December 14, 2018.
Basis for approval
The drug showed positive results in both a Phase 3 and Phase 1/2 study, warranting its approval. These studies were both placebo-controlled. In the Phase 3 study, platelet response increased by 71% for those given Nplate versus just 30% for those given placebo. Durable platelet response was also measured and also showed a positive response for Nplate (52% vs. 10% in placebo). Adverse effects in both the Phase 3 and Phase 1/2 study occurred less than 25% of the time. The effects documented were oropharyngeal pain, contusion, and upper respiratory tract infection.
This approval should give ITP patients more options. Nplate’s ability to maintain safe platelet levels will not only benefit patients medically, it will give them and their families a greater peace of mind. The condition, when left untreated, can cause families a lot of anxiety due to its spontaneity. Hopefully, this new approval will give more ITP children and their parents an improved quality of life.
Nplate also has approval in Canada, Japan, and Australia, as well as 64 other countries.
You can read more about Nplate and the research that brought its approval here.