Potential Treatment for Niemann-Pick Disease is Granted Orphan Drug Designation in the EU

According to a story from rdmag.com, the biopharmaceutical company IntraBio Inc., recently announced that its IB1000 series of investigational therapeutic compounds have been granted Orphan Medicinal Drug Designation as a therapy for Niemann-Pick disease. IntraBio is dedicated to the development of innovative therapeutics for the treatment of both rare and common neurodegenerative diseases.

About Niemann-Pick Disease Type C

Niemann-Pick disease type C is a form of lysosomal storage disease which is characterized by a deficiency not in an enzyme, but most typically in a type of transporter protein that prevents water soluble molecules from moving within a cell. It is caused by mutations of either the NPC1 or NPC2 gene. There is broad disparity in the severity and presentation of symptoms in Niemann-Pick disease type C, making symptoms an unreliable method for diagnosis. They may appear in childhood or as late as a patient’s sixth decade of life. Such symptoms include spleen and/or liver enlargement, jaundice, severe depression, ataxia, epilepsy, difficulty speaking and swallowing, dystonia, poor muscle tone, bipolar disorder, microcephaly, progressive loss of hearing, progressive dementia, and psychosis. Most treatment is supportive, but there are some medicines that can delay disease progression and prolong life. Lifespan is connected to the onset of symptoms, with those with the earliest symptoms usually dying sooner. To learn more about Niemann-Pick disease type C, click here.

Encouraging Findings

The data so far for the IB1000 series has been encouraging. These experimental drugs have performed well in compassionate use clinical trials. They appear to be capable of reducing symptoms and providing protection to the nervous system. The compounds also appear to be relatively safe.

Orphan Drug Designation in the EU

The orphan designation in the EU is modeled closely off of the comparable designation that is used by the FDA in the US. Benefits of the designation include the waiving of fees related to the submission of the marketing application, a ten year period of market exclusivity if the drug is approved successfully, and eligibility for a single marketing application that is automatically valid in all EU member countries upon approval.

Aside from Niemann-Pick disease type C, IntraBio is also prioritizing the development of the IB1000 series for Sandhoff disease, Tay-Sachs disease, and cerebellar ataxia. However, these experimental compounds could be viable for many other diseases as well.


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