Drug discovery
3
drugs
With orphan designations
Overview
Benign schwannomas are slow-growing, encapsulated tumors arising from Schwann cells of peripheral nerves, most commonly affecting the vestibular nerve (vestibular schwannoma) or spinal nerves [1][4][16]. Symptoms include localized pain, neurological deficits (numbness, weakness), and cranial nerve dysfunction (hearing loss, tinnitus, imbalance) [6][13][17]. Diagnosis relies on MRI/CT imaging and histopathological confirmation via biopsy [1][16]. Management options include surveillance for asymptomatic tumors, microsurgical resection for symptomatic cases, and stereotactic radiosurgery for inoperable tumors [1][13][19]. Prognosis is excellent post-resection, though rare malignant transformation (<2.5%) occurs, particularly in neurofibromatosis type 2 (NF2) or radiation-exposed patients [11][19][16].
Population
Peak incidence: 65–74 years (spinal) and 45–64 years (vestibular) [2][12].
Male predominance in spinal cases (IRR 0.86 vs. females) [2]; vestibular cases show slight female preference (52.6%) [7].
Associated with NF2 (bilateral vestibular tumors) and schwannomatosis (multiple peripheral tumors) [3][5][16].
Burden
Functional morbidity: Hearing loss (68.8%), facial palsy (82.2%), and balance disorders (91.8%) in vestibular cases [17].
Economic impact: Lifelong surveillance (MRI every 6–12 months) and surgical costs [1][13].
Malignant risk: <2.5% develop MPNSTs, particularly in NF2 or post-radiation cases [11][19].
Therapies
Categories: rare neoplastic diseases, rare neurological diseases
Drug Discovery Landscape
Drug | Therapy type | Regulator | Orphan designation | Approval | Sponsor |
|---|---|---|---|---|---|
Salmonella enterica, subsp. enterica, serovar Typhimurium, strain YS1646, live | other | EMA | 2022-07-18 | — | Premier Research Group S.L. |
Live attenuated Salmonella enterica Serovar Typhimurium (S. Typhimurium) | gene therapies | FDA | 2022-05-10 | — | Mulberry Biotherapeutics, Inc. |
N-hydroxy-4-(3-methyl-2-(S)phenyl-butyrylamino)benzamide | small molecules | EMA | 2012-06-06 | — | Propharma Group The Netherlands B.V. |