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news.The US Food and Drug Administration (FDA) has granted orphan drug designation for Protalex's lead product, PRTX-100, for the treatment of immune thrombocytopenia (ITP), an autoimmune-mediated condition.
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Earlier, the agency has accepted the company’s investigational new drug (IND) application for a Phase I/II open-label, dose-escalating trial of PRTX-100 in adults with persistent/chronic ITP.
The company intends to enroll its first patient in an ITP trial of PRTX-100, a highly purified form of Staphylococcal Protein A, in the third quarter of this year.
ITP is characterized by bruising and increased bleeding as a result of immune-mediated accelerated destruction of platelets and impaired production of platelets.
Protalex chief medical officer Dr William Gannon said: "The FDA’s timely approval of our request for orphan drug designation for PRTX-100 to treat ITP is a key milestone that supports our broader strategy of bringing this potentially life-saving therapy to patients with ITP.
"We look forward to enrolling patients in our Phase I/II clinical trials of PRTX-100 to treat patients with ITP and expect to have top-line data in 2016."
Nplate (romiplostin) and Promacta (eltrombopag) are the two most recently approved drugs used to treat ITP. Both the drugs are said to increase the production of platelets but do not appear to affect the underlying platelet destruction process.
The company said that in contrast, pre-clinical data show that PRTX-100 may have the potential to treat ITP by reducing the immune-mediated destruction of the platelets.
In addition, PRTX-100 has established an acceptable safety profile based on data from patients treated in five clinical trials including patients with rheumatoid arthritis (RA).