EMA Grants Orphan Drug Designation to NIDO-361 for Kennedy's Disease

Nido Biosciences, a biopharmaceutical company developing medicines to treat debilitating neurological diseases with precision, today announced that the European Medicines Agency has granted an Orphan Drug Designation for its lead clinical candidate, NIDO-361. This novel small molecule is designed to treat the spinal and bulbar muscular atrophy, also known as Kennedy’s disease; the designation has been made after the company reported full enrollment of the Phase 2 study for the medicine.

Publish Date: 22-10-2024 Source: Nido Biosciences

Kennedy’s disease, called bulbospinal muscular atrophy (BSMA), is a rare genetic disorder following an X-linked recessive inheritance pattern characterized by the degeneration of motor neurons leading to muscle wasting in the proximal and bulbar regions. BSMA is caused by an unstable expansion of a CAG triplet repeat (40-62 repeats) in exon 1 of the androgen receptor (AR) gene located on chromosome Xq11-12. The onset of symptoms typically occurs between the ages of 30 and 60 years, and initial clinical manifestations may include tremors, muscle cramps, twitching, fatigue, and slurred speech. With disease progression, patients may experience weakness and wasting of the limb and bulbar muscles, resulting in dysarthria, dysphonia, hanging jaw, tongue wasting, chewing difficulty, and impaired mobility. The disease may be misdiagnosed initially as hereditary spastic paraplegia, spinocerebellar ataxia, other motor neuron diseases, myopathies, neuropathies, or cervical spondylosis. The progression of BSMA is slow, with only one-third of patients requiring a wheelchair 20 years after diagnosis. The prognosis for BSMA is generally favorable, with only a slight decrease in life expectancy.

The estimated incidence of Kennedy’s disease in the USA is approximately 1 case in 40,000 men.

However, the current Kennedy’s Disease treatment market share, market uptake, and attribute analysis concerning the most potential emerging therapies (OAV101, AJ201, etc..) has been provided under the market outlook section of the study covering 8 MM countries; The United States, EU5 (Germany, Spain, France, Italy, UK) Japan and China.

In terms of pharmacologic therapies, several pharmaceutical products are being approved and under different phases of development for the Kennedy’s Disease treatment. The key companies in the advanced development stage are Novartis Pharmaceuticals, AnnJi Pharmaceutical Co., Ltd., etc..

Based on solid domain and business knowledge, Thelansis Knowledge Partners has published the market outlook forecast report on Kennedy’s Disease to provide a clear understanding of disease area background, epidemiology, current and future competitions, the country-specific standard of care, and the complete market forecast for 2023 to 2033.

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