Blueprint Medicines Submits New Drug Application to the FDA to Treat Thyroid Cancer


A recent article in BioSpace reported that Blueprint Medicines Corp. has submitted a New Drug Application (NDA) to the FDA for the investigational drug pralsetinib with an action date of November 23, 2020. The drug has been developed to treat patients who are diagnosed with RET fusion-positive thyroid cancers or RET mutated medullary thyroid cancer.

The NDA was submitted through an FDA program designed for efficiency, safety, quality, and ensuring that newly-developed treatments are made available as quickly as possible.

Earlier this year Blueprint Medicines reported that the FDA and the EMA have accepted Blueprint’s application to market pralsetinib.

Genetically Altered Solid Tumors

RET mutations and fusions (a RET gene fused to another gene) have been implicated in non-small cell lung cancer (NSCLC) and medullary thyroid cancer (MTC). The RET protein is altered by a change in the RET gene in about one in seventy-five NSCLCs. RET is an acronym for REarranged during Transfection (altering).

Gene fusion occurs most often while RET fusions generally occur in a form of NSCLC called adenocarcinomas. RET alterations have also been found in pancreatic, breast, colorectal, and various other cancers.

About Pralsetinib

Pralsetinib is a precision medical treatment that is taken once each day. It is designed for fusion-positive NSCLC patients with evidence of cancer progression after they had been treated with platinum-based chemotherapy.

Pralsetinib’s second target is RET mutation-positive medullary thyroid cancer for which no alternative treatments exist.

The research team at Blueprint Medicines designed pralsetinib. The drug has demonstrated potency against RET fusions and mutations in preclinical evaluations.

Further, pralsetinib has shown that it can suppress primary and secondary mutations. This is an indication that it is capable of preventing therapy resistance.

 About Blueprint Medicines

Blueprint Medicines, specializing in precision therapy, is primarily focused on rare diseases, genomically defined cancers, and cancer immunotherapy. The company has entered into an exclusive license agreement with CStone Pharmaceuticals to develop and commercialize pralsetinib along with several other products. The agreement covers new drug development in Hong Kong, Taiwan, Macau, and China’s mainland.

However, Blueprint has retained commercial and development rights in all other jurisdictions.

Looking Toward the Future

Dr. Vivek Subbiah, of MD Anderson’s Investigational Therapeutics Department, referred to results from the ongoing phase 1/2 clinical trial (NCT03037385) demonstrating that pralsetinib has broad antitumor activity across solid tumors that host RET alterations.

Dr. Subbiah concluded by saying that genome-driven oncology has had a positive impact on many hematologic and solid malignancies.

What are your thoughts about this new precision medical treatment for cancers that to date have been difficult to treat? Share your stories, thoughts, and hopes with the Patient Worthy community!

Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia four years ago. He was treated with a methylating agent While he was being treated with a hypomethylating agent, Rose researched investigational drugs being developed to treat relapsed/refractory AML.

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