Research & Development
 

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 on an annual basis further extensions are granted by the US FDA for 12 month periods.

Daval is currently in the process of applying for Orphan Drug Designation with the European Medicines Agency (EMA) and with the Food & Drug Administration (FDA) for Scleroderma.

Daval is also applying for Early Access in the UK via ABPI for Scleroderma.

“Special” status has been given to AIMSPRO in Australia and the UK

In October 2007 AIMSPRO became available in Australia under Categories A and B of the TGA’s Special Access Scheme.

In September 2005 AIMSPRO was granted permission, by the MHRA, to be manufactured as a “Special”. This designation allows UK doctors to prescribe the therapeutic on a Named Patient/Compassionate Use basis.

Good Clinical Practice and Good Manufacturing Practice audits are carried out regularly

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).

Manufacturing facilities have been inspected & audited by the MHRA and the TGA in their respective countries.

Health Protection Agency (UK) are reviewing the stability of product on an ongoing basis from a manufacturing standpoint.

Functional assay process is being conducted independently on an on-going basis with two independent commercial laboratories (Finland, USA).

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.