Regulatory Status

Orphan Drug Designations

Use in a small number of patients with various leukodystrophies led to the awarding of an initial Orphan Drug designation for AIMSPRO by the Therapeutic Goods Administration (TGA) in Canberra, Australia for the treatment of Krabbe disease (also known as globoid cell leukodystrophy) in infants.

On 15th May 2009 the TGA awarded AIMSPRO a second Orphan Drug designation for the treatment of Amyotrophic Lateral Sclerosis (ALS - the most common form of Motor Neuron Disease, also known as Lou Gehrig’s Disease).

AIMSPRO was awarded an Orphan Drug Designation by the Food & Drugs Administration (FDA) in the United States for the treatment of ALS, with effect from 28th October 2009. Following submission of additional supporting data, a further extension was granted recently by the US FDA for 12 months.

Daval has filed an application for Orphan Drug Designation with the European Medicines Agency (EMA) for Systemic Sclerosis, which has been declared valid and approval is expected in June 2012.

Trial Regulatory Compliance

All clinical trials undertaken, where Daval has been the sponsor, have been granted local Ethics Committee approvals.

Daval was inspected by the MHRA in December 2009 and has demonstrated to the agency’s satisfaction that the company complies with Good Clinical Practice (GCP). In addition, all production and sourcing are fully GMP and GDP compliant.

Authorisations

Ceremben has a Minor Use permit (Permit Number PER12341 - awarded in September 2010) from the Australian Pesticides and Veterinary Medicines Authority for the sale of the product to Veterinary Surgeons in Australia for the treatment of endotoxaemia in horses.