In short
Nomlabofusp is an investigational protein-based medicine being studied for Friedreich's ataxia, a rare inherited disease that affects movement and coordination. The registry lists one authorised Phase 3 trial with a planned enrollment of 150 adults and children. The trial compares nomlabofusp given by subcutaneous injection against placebo over 72 weeks, measuring change on the Modified Friedreich's Ataxia Rating Scale (mFARS).
Key points
- Nomlabofusp is an investigational protein-based substance with no marketing authorisation in the product records.
- The single registered trial is a Phase 3 study in adults and children with Friedreich's ataxia.
- The trial plans to enroll 150 participants and is currently authorised.
- Nomlabofusp is given as a subcutaneous injection at a concentration of 50 mg/ml.
- The main outcome measure is change on the Modified Friedreich's Ataxia Rating Scale (mFARS) at week 72.
- Nomlabofusp has orphan designation for Friedreich's ataxia, a rare disease.
What is nomlabofusp?
Nomlabofusp is an investigational protein-based substance, meaning it is made from proteins rather than being a small chemical molecule. It is being developed by Larimar Therapeutics, Inc. The product records list it as a solution for injection with no marketing authorisation, which means it is not yet approved as a medicine. Nomlabofusp has received orphan designation, a status given to treatments for rare diseases such as Friedreich's ataxia. The substance has also been studied under the development code CTI-1601.
How does nomlabofusp work?
Friedreich's ataxia results from a deficiency of the mitochondrial protein frataxin (FXN). Nomlabofusp is designed to deliver frataxin to the body, aiming to address the underlying protein deficiency rather than only treating symptoms. The registry data describe Friedreich's ataxia as a rare, progressive, multisystem disease with an estimated incidence of 1 in 29,000 people and a carrier frequency of about 1 in 85.
Which conditions is nomlabofusp studied for?
- Friedreich's ataxia — the most common inherited ataxia in humans, a rare and progressive disease affecting the nervous system and other parts of the body, caused by a deficiency of the frataxin protein.
How is nomlabofusp given?
Nomlabofusp is given as a subcutaneous injection, meaning it is injected under the skin. The product record lists it as a solution for injection at a concentration of 50 mg per millilitre.
What do the clinical trials show?
The registry contains one trial of nomlabofusp, a Phase 3 study that is currently authorised. The trial is designed to learn whether nomlabofusp improves movement ability in adults and children with Friedreich's ataxia compared with placebo.
The trial plans to enroll 150 participants. Its main outcome measure is the difference between participant groups on the Modified Friedreich's Ataxia Rating Scale (mFARS) total score at study week 72 compared with baseline. The mFARS is a scale used to assess movement and coordination in people with Friedreich's ataxia.
A published earlier study reported on the safety, pharmacokinetics and pharmacodynamics of nomlabofusp, previously known as CTI-1601, in Friedreich's ataxia.
What side effects are known?
The published study on nomlabofusp (CTI-1601) in Friedreich's ataxia reports on its safety profile, but the provided source material does not list specific side effects in detail.