At the very end of March 2021, specialty pharmaceuticals company JCR Pharmaceuticals Co., Ltd. (“JCR”) shared that its investigational drug candidate JR-171 received Orphan Drug designation from the European Medicines Agency (EMA). The medicine, which is able to cross the blood-brain barrier to more effectively deliver treatment, is designed for patients with mucopolysaccharidosis type I (MPS I).
So what is JR-171? This recombinant alpha-L-iduronidase is specially formed to cross the blood-brain barrier. Altogether, JR-171 was developed using JCR’s proprietary J-Brain Cargo technology. Currently, other forms of treatment do exist for MPS I, such as intravenously administered enzyme therapies. However, while these can alter disease progression and provide symptom relief, current therapies fail to address the neurological and brain-related symptoms. By crossing the blood-brain barrier, JR-171 offers the chance to fulfill an unmet need and provide additional symptom relief to patients.
At the moment, JCR is evaluating JR-171 for patients with MPS I in a Phase 1/2 clinical trial. Interested in learning more about the trial, which is enrolling patients from across the globe? Click here.
In February 2021, JR-171 was granted Orphan Drug designation by the FDA. In the United States, Orphan Drug status is given to drugs or biologics centered around treating rare diseases. When in the US, rare diseases are defined as those affecting 200,000 people or less. Now, JR-171 also received Orphan Drug designation from the EMA. According to the EMA:
About 30 million people living in the European Union (EU) suffer from a rare disease. The European Medicines Agency (EMA) plays a central role in facilitating the development and authorisation of medicines for rare diseases, which are termed ‘orphan medicines’ in the medical world.
To be considered a rare disease in the European Union (EU), a condition must not affect more than 5 in every 10,000 individuals. Once a drug is granted Orphan Drug status, there are also benefits conferred for the drug developer, including:
- 10 years of market exclusivity
- Protocol assistance
- Centralized authorization procedure
MPS I (Hurler Syndrome)
Also known as Hurler syndrome, mucopolysaccharidosis type I (MPS I) is a rare metabolic disease of which there are three forms. Patients do not have enough lysosomal alpha-L-iduronidase enzyme, preventing them from breaking down mucopolysaccharides. As described by the National MPS Society:
Mucopolysaccharides are chains of sugar molecules used to build connective tissues in the body. “Muco” refers to the thick jelly-like consistency of the molecules; “poly” means many; “saccharide” is a general term for a sugar molecule.
When these mucopolysaccharides build up throughout the body, they can cause widespread health effects. Altogether, around 1 in every 100,000 newborns has severe MPS I, while 1 in every 500,000 has milder MPS I. Typically, symptoms begin to appear between ages 3-8. Once symptoms do appear, they include:
- Progressive intellectual delays
- Joint stiffness
- Corneal clouding
- Short, broad hands with curved fingers
- Spleen and liver enlargement
- Sleep apnea
- Abdominal hernias
- Stunted growth
- Spinal cord compression
- Heart disease