ICYMI: First Drug to be Approved for Neurofibromatosis Type 1 and Plexiform Neurofibromas (NF1-PN)

 

According to a recent announcement by AstraZeneca, the U.S. FDA granted its approval for Koselugo (selumetinib). This is the first drug to be approved for the treatment of children with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN). PNs occur inside nerve sheaths in the form of bulging masses in skin folds and connective tissue.

The Phase II SPRINT Trial

The approval was based on the Phase II SPRINT Stratum 1 trial that showed selumetinib reduced inoperable tumors, reduced pain, improved function, and improved overall quality of the children’s lives after treatment.

A total of fifty patients participated in the Phase II trial. Researchers assessed outcomes according to each individual patient’s tumor-related symptoms. This approach had not been used in previous NF1 clinical trials.

Results

The Overall Response Rate (ORR) was defined as a percentage of patients who experienced a complete response or experienced a minimum of twenty percent reduction in PN (inoperable tumor) volume.

ORR for the Phase II trial was sixty-six percent (partial response) with eighty-two percent of these patients maintaining the response for one year or more. No patients had their tumors completely disappear.

About Koselugo (selumetinib)

Koselugo, a kinase inhibitor, blocks critical protein kinase enzymes, thus preventing the growth of tumor cells.

The drug has been developed and approved specifically for patients with inoperable plexiform neurofibromas (PN). These tumors can be found in many areas of the body such as extremities, face, around the spine, or deep within the body.

A total of fifty patients participated in the Phase II trial. Researchers assessed outcomes according to each individual patient’s tumor-related symptoms. This approach had not been used in previous NF1 clinical trials.

Adverse Responses to Selumetinib

The most common side effects to the drug include vomiting, diarrhea, abdominal pain, rash, fever, musculoskeletal pain, and fatigue.

Patients may also experience inflammation of the mouth and lips (stomatitis), infections of the skin surrounding toe or fingernails (paronychia) and itching.

More serious side effects may involve heart failure or ocular toxicity, which is acute and chronic damage to the eye.

Researchers discovered during animal studies of selumetinib that the drug may have harmful effects on newborn babies if it is given to pregnant women.

About FN1-PN

NF1 affects approximately one out of every three thousand infants. About thirty to fifty percent of NF1 patients experience PN tumors.

PNs may cause symptoms such as motor, airway, bladder, or bowel dysfunction, as well as possible disfigurement.

FDA Designations

The FDA granted AstraZeneca Pharmaceuticals the Rare Pediatric Disease designation for treatment of pediatric NF1.

About AstraZeneca

AstraZeneca Pharmaceuticals LP is a global biopharmaceutical company. It is based in Cambridge, UK and operates in over 100 countries. Its medicines are used by millions of patients worldwide. The company’s focus is on three main therapy areas: oncology, cardiovascular & metabolic disease. AstraZeneca also works with biotech research institutions on emerging technologies such as modified RNA and CRISPR genome editing.


What are your thoughts about this new drug that brings improved quality of life to patients? 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.

Share this post

Share on facebook
Share on google
Share on twitter
Share on linkedin
Share on pinterest
Share on print
Share on email
Close Menu