Partnership for treatment of Niemann-Pick Type C


Medical Need and CTD Holdings enter into a partnership for treatment of Niemann-Pick Type C disease

Medical Need today announced that it has entered into an exclusive supply and distribution agreement with the Florida based CTD Holdings relating to their product Trappsol® Cyclo (hydroxypropyl beta cyclodextrin (HPBC)).

The product is under development for the treatment of Niemann-Pick disease Type C (NPC). NPC, a lysosomal storage disorder, is a severe inborn genetic defect of the cholesterol metabolism, presenting in childhood and leading to dysfunction of the liver and lungs, neurodegeneration and premature death. Trappsol® Cyclo received an orphan drug designation for the treatment of NPC in the US in 2011 and has been used to treat NPC patients in the US, Europe and other parts of the world on a named patient basis under special permission by national regulatory authorities.

Under the agreement, Medical Need is awarded the exclusive rights to Trappsol® Cyclo in Europe, including all EU member states, Norway, Iceland, Greenland, Switzerland, Turkey, the Balkans, Tunisia and Algeria, and will be responsible for the registration, distribution, marketing and sale of the product in the territory. Initially, the product will on request be provided on a named patient basis.

“Trappsol® Cyclo is a very interesting product under development for a rare disease with significant unmet medical needs”, said Dr. Peder Walberg, CEO of Medical Need, and continued: “We are very excited to collaborate with CTD in developing this important therapy for the benefit of the NPC patients in Europe.”

About Niemann-Pick disease type C (NPC)

Niemann-Pick disease is an inborn genetic error of cholesterol metabolism, belonging to the group of lysosomal storage disorders. It is caused by a mutation in one of two key proteins for transportation of cholesterol within the cell (NPC1 and NPC2), and leads to the accumulation of unesterified cholesterol in the lysosomes of almost all cells of the body. Cholesterol is a very important and normally tightly regulated molecule and excess accumulation has detrimental effects on cell function. NPC normally presents in childhood and eventually leads to hepatic and pulmonary dysfunction, neurodegeneration and premature death.

About Trappsol® Cyclo

Trappsol® Cyclo (hydroxypropyl beta cyclodextrin (HPBC)) is a cGMP manufactured, endotoxin controlled, ready to use solution for infusion.

Cyclodextrins are naturally occurring rings of sugar molecules derived from starch. Their predominant property is their ability to host hydrophobic molecules and improve their solubility. Cyclodextrins are presently used as an excipient in several approved pharmaceuticals and are classified as GRAS (Generally Regarded as Safe) by the FDA.

HPBC is well known for its ability to solubilize cholesterol and has shown therapeutic efficacy in animal models of NPC. Trappsol® Cyclo has been used to treat patients with NPC in the US, Europe and other parts of the world under special permission from national regulatory authorities, on a so called named patient basis. The product was awarded orphan drug status in the US in 2011.

 

About CTD Holdings

CTD Holdings is a Florida based company, specializing in the manufacture, development and marketing of cyclodextrins. The company is listed on the US OTC market. For more information please visit www.ctd-holdings.com.