FDA Grants Orphan Drug Designation for Acute Radiation Syndrome Drug

The Food and Drug Administration (FDA) has granted Orphan Drug Designation for the novel drug, NT-17 (efineptakin alfa), to treat acute radiation syndrome (ARS), according to a news release.

Acute radiation syndrome, as the National Cancer Institute explained, is a serious illness that is caused by exposure to high amounts of radiation during a short amount of time. This rare illness also may cause severe damage to the immune system and bone marrow, the news release stated.

NT-17 is a drug that is currently being analyzed for oncologic and immunologic uses, notably where T-cell amplification and increased functionality could offer clinical benefits, according to the news release. The drug is also being evaluated in clinical trials for solid tumors and as a vaccine adjuvant.

The novel drug is reported to be an effective treatment for acute radiation syndrome that may have the potential to help recover and improve the immune response. According to the news release, NT-17 has been effective in a clinical setting, as it consistently boosted T cell count. It also reportedly demonstrated tolerability and safety.

“Obtaining the Orphan Drug Designation from the FDA is an important acknowledgment of the immense potential that NT-I7 holds in providing a beacon of hope for the treatment of ARS,” Se Hwan Yang, president and chief executive officer of NeoImmuneTech, the manufacturer of NT-17, said in the news release.

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Symptoms of ARS can appear following exposure or over time, in which symptoms may “come and go,” the National Cancer Institute noted. In particular, the severity of the symptoms depends on the amount of radiation that a person was exposed to and absorbed, Mayo Clinic stated.

Initial symptoms are typically nausea and vomiting, in which the onset of these symptoms may help determine how much radiation a person has absorbed. With severe exposure, symptoms of ARS can arise from minutes to days following the exposure.

The symptoms following initial symptoms may include diarrhea, fever, hair loss, weakness/fatigue, infections, headache, low blood pressure, dizziness and bloody vomit or stools from internal bleeding.

Side effects of radiation damage can also appear as skin damage, similar to a bad sunburn. This can also lead to symptoms of swelling, redness and itching days or even weeks later, according to the Centers for Disease Control and Prevention.

In 2018, the FDA approved Leukine (sargramostim) for patients who were exposed to myelosuppressive doses of radiation, which is a subsyndrome of ARS.

The FDA granted an orphan drug designation for NT-17 in 2022 to treat patients with glioblastoma multiforme, known to be a fast-growing and aggressive brain tumor.

Preliminary data from a trial regarding NT-17 for glioblastoma multiforme demonstrated significant results in progression-free survival (the length of time during and after treatment when a patient lives with disease but it does not worsen) and overall survival (the length of time from diagnosis or the start of treatment when a patient is still alive).

In late 2022, the treatment combination of NT-17 and Keytruda (pembrolizumab) established a higher objective response rate (the percentage of patients who have a partial or complete response to treatment within a certain period) for some patients with microsatellite stable colorectal cancer and liver cancer without liver metastasis, demonstrated by data from a phase 1/2 trial.

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