The European Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion regarding Raptor Pharmaceutical Corporation’s drug, Procysbi, for treating proven nephropathic cystinosis, a rare inherited genetic disorder that causes irreversible tissue damage, organ failure and premature death.

On September 20th, 2010, Procysbi was designated as an orphan medicinal product in Europe, because it targets a rare condition or disease.

Cystinosis is a very uncommon genetic condition that affects approximately 3,000 people worldwide. If not treated early on in childhood, the condition is fatal. Cystinosis causes an accumulation of cystine in every cell in the body. Cystine is a protein building block. When too much cystine builds up, it leads to kidney problems, which in turn causes the body too lose too much glucose, salts and protein through urine. Cystinosis is more common among white, blue-eyed people of European descent.

People with cystinosis may experience slow body growth, small stature, worsening renal failure and weak bones. Nephropathic cystinosis is the most severe of the three types of cystinosis.

The CHMP’s green light does not mean Procysbi (cysteamine bitartrate) 25mg and 75mg gastro-resistant hard capsules have been approved – final approval is decided by the European Commission (EC). However, the EC nearly always goes along with CHMP’s recommendations. An EC’s decision regarding drug approvals covers 27 EU countries, including Iceland and Norway.

Procysbi

CHMP wrote “The approved indication is: “Procysbi is indicated for the treatment of proven nephropathic cystinosis. Cysteamine reduces cystine accumulation in some cells (e.g. leukocytes, muscle and liver cells) of nephropathic cystinosis patients and, when treatment is started early, it delays the development of renal failure.”

“The CHMP, on the basis of quality, safety and efficacy data submitted, considers there to be a favourable benefit-to-risk balance for Procysbi and therefore recommends the granting of the marketing authorisation.”

CHMP proposes that only doctors experienced in treating cystinosis prescribe Procysbi.
Read more here.