According to a recent press release from biopharmaceutical company NFlection Therapeutics, Inc. (“NFlection”), the FDA granted Orphan Drug designation to the company’s drug candidate NFX-179. Altogether, this treatment is designed for patients with cutaneous neurofibromatosis type 1 (NF1). Orphan Drug designation is granted to drugs or biologics intended to treat patients with rare or underserved conditions, defined as those affecting under 200,000 American citizens. Because NFX-179 received this status, NFlection also earned fee waivers, tax credits, increased regulatory assistance, and 7 years of market exclusivity if the drug is approved.
According to NFlection, the company is currently developing:
A topical gel containing NFX-179, a proprietary ‘soft’ (metabolically labile) MEK inhibitor for the reduction of tumor burden of persistently developing cutaneous neurofibromas (cNF) in neurofibromatosis type 1 (NF1). MEK inhibitors block RAS pathway signaling downstream of the neurofibromin 1 mutation and represent a promising targeted therapeutic.
Currently, NFlection is evaluating NFX-179 in a series of Phase 2 clinical trials. This treatment has the potential to limit side effects often associated with MEK inhibitors and provide more effective, targeted treatments.
Altogether, there are three forms of neurofibromatosis, a rare genetic disorder which affects the nervous system, which all have different manifestations. In each case, neurofibromatosis causes benign or malignant tumors to grow on healthy nerve tissue. NF1 gene mutations cause neurofibromatosis type 1 (NF1), which often begins in childhood and causes skin issues and bone deformities. Next, NF2 gene mutations cause neurofibromatosis type 2 (NF2), in which symptoms, including hearing issues, begin in the teenage years. Finally, SMARCB1 or NF2 gene mutations cause schwannomatosis, the rarest form, which often manifests in intense pain.
Symptoms associated with cutaneous NF1 include:
- Bone deformities
- Short stature
- Neurofibromas (soft bumps on or under the skin)
- Tiny bumps on the eyes’ irises
- Flat, light brown spots on the skin
- Freckling around the armpits or groin
- Learning or cognitive disabilities
Next, patients with NF2 may experience poor balance, hearing loss, tinnitus (ringing in the ears), or headaches. Finally, symptoms associated with schwannomatosis include tumors on the skull, spinal nerves, or peripheral nerves; muscle numbness or weakness; and muscle loss.