Pricing negotiations for Koselugo’s reimb start after 2 yrs

treatment for neurofibromatosis

Passes DREC meeting in September after 3 tries

Attention focused on the potential birth of the virtually only treatment option

Koselugo, which is a treatment for pediatric patients with neurofibromatosis, has entered its last gateway to receiving reimbursement in Korea.

 

According to industry sources, AstraZeneca has recently started drug pricing negotiations with the National Health Insurance Service to set the insurance price for its new neurofibromatosis drug, Koselugo.

 

Whether Koselugo, which passed the Health Insurance Review and Assessment Service’s Drug Reimbursement Evaluation Committee review on the 7th of last month after 3 attempts, will be able to settle on a final drug price during negotiations and be listed for reimbursement remains to be seen.

 

This drug has remained non-reimbursed for 2 years now.

 

It has only been able to enter the NHIS negotiation stage after much trouble.

 

Koselugo, which was designated as the first drug subject to fast-track review in October 2020, obtained marketing authorization from the Ministry of Food and Drug Safety on May 28, 2021, and submitted an application for its reimbursement in May 2021, but failed to pass the DREC gateway in March last year.

 

The company quickly supplemented the data and restarted discussions in April this year, bringing the situation to what it has become now.

 

Until now, patients had to rely only on symptomatic treatment for neurofibromatosis due to the lack of an appropriate treatment option.

 

Neurofibromatosis is a rare disease.

 

85% of the patients with neurofibromatosis have neurofibromatosis type 1 (NF1), which is caused by a mutation in the neurofibromin tumor suppressor gene located on chromosome 17.

 

The incidence of NF1 is approximately 1 in 3,000.

 

Its first symptom is café-au-lait spots 1 to 3 centimeters in diameter early in life.

 

Since then, the patients have experienced Optic nerve gliomas (brain tumor) at age 6, and scoliosis around age 6-10.

 

In adulthood, lisch nodules, or iris hamartomas, occur predominantly in patients with NF1.

 

If possible, treatment includes surgical removal of affected sites or chemotherapy and radiation therapy.

 

However, most recur even after surgery, and as the patient must undergo a major operation, its treatment puts an immense burden on both the medical staff and the patient.

 

Recurrence is even more frequent among pediatric patients, which means the patients must live with painkillers and often suffer from speech and movement disorders even after receiving several operations.

 

Koselugo was jointly developed by AstraZeneca and MSD.

 

The drug blocks the activation of MEK to inhibit the growth of cell lines.

 

In the Phase II SPRINT study that became the basis for Koselugo’s approval, Koselugo reduced tumor size by over 20% in 68% of the patients who received Koselugo and achieved its primary endpoint of ORR.

 

Also, 82% of the patients who showed a partial response had sustained responses lasting at least 12 months.

 

In contrast to the non-treated patients, half of whom experienced disease progression 1.5 years after diagnosis, only 15% of patients using Koselugo showed disease progression at year 3.