FDA Approvals in 2018


Newly born children might suffer from Immune Thrombocytopenia. It is a condition in which platelets are destroyed by the immune system which results in bruises and bleeding to occur easily. Corticosteroids and removing spleen didn’t show any usefulness so a new drug has been identified that is Nplate. It has been approved by FDA this year.

Nplate (romiplostim) is an injectable protein medication. It has been identified as a treatment for 1-year children who are suffering from Immune Thrombocytopenia for 6 months and haven’t shown a response to any other treatment. This approval was made after 2 double-blind, Placebo-controlled trials. The first trial had 62 patients and the other had 22 patients. In the first trials, patients were either treated by Nplate or Placebo. Response rates were 52% and 10% respectively for both groups. 71% versus 20% was an overall response from both groups.

In case of the randomized trial of 22 patients, they received Nplate or Placebo. Among them, 88% showed a constructive response with an increased platelet count within 2 weeks. Side effects were also visible in more than 25% of those patients. So overall, romiplostim is effective for children with ITP.

The second approval by FDA in 2018 is a biosimilar product identical copy of the original product but is produced by another company. Herceptin (trastuzumab, Genentech) was used to treat HER-2 overexpressing in breast cancer. A biosimilar has been prepared for it that is Herzuma (trastuzumab pkrb). Trastuzumab-pkrb is a HER/2 neu receptor antagonist. Indications of risks involved with the use of both of these drugs are given in a box for these drugs. Possibly cardiomyopathy, infusion reactions, pulmonary toxicity, and embryo-fetal toxicity are expected.

Drugs have adverse effects along treatment. Diarrhea, nausea, chills, fever, infections, congestive heart failure are side effects of Herzuma. In some cases worsening of chemotherapy might occur and it causes neutropenia.