We know they will get Orphan Drug Status because Austrianova already received it once before.....
EMEA previously granted Orphan Drug Status for this product which gave:
Free Protocol Advice
“fast track” thro‘ further clinical trial design together with EMEA
Up to ten years market exclusivity
Successful clinical trials using the Cell-in-a-Box® principal:
Use of encapsulated cells for cancer cell therapy from genetically modified cells over-expressing a chemo-activating enzyme to allow lower doses of chemotherapy to be used more efficaciously without the usual debilitating side effects. Shown to be safe and efficacious in early phase clinical trials
Product:
A cell based therapeutic consisting of cells genetically modified to over-express cytochrome P450 encapsulated in biologically inert cellulose sulphate polymers
– one “off the shelf “ product for all patients
Mechanism of Action:
The encapsulated cells are placed, using supraselective catheterisation, into blood vessels leading to the tumor where they locally convert a chemotherapeutic prodrug to its tumor toxic product
Proven Safety in the clinic:
Phase l/II Trial design
Advanced non-resectable pancreatic cancer
14 patients treated
One dose of 300 microcapsules
Delivered by catheter into tumor artery
1/3 dose chemotherapy
Therapeutic Effect shown through Tumor Reductions:
A second, confirmatory trial involving more clinical centers was undertaken. The overall Results of Clinical Trial:
Safety
Proven safety record in all 27 patients
No obvious immune or inflammatory response to cells or capsule material
Efficacy
Double survival rate over gold standard
Tumor shrinkage
Improvement in Quality of Life
Lower dose ifosfamide gives a similar survival benefit but fewer side effects
In addition
the treatment strategy and technology recognized by regulatory authorities EMEA, TGA, FDA, HSA
EMEA previously granted Orphan Drug Status for this product which gave:
Free Protocol Advice
“fast track” thro‘ further clinical trial design together with EMEA
Up to ten years market exclusivity
Results published in peer-reviewed journals such as Lancet, Cancer Therapy, Gastroenterology
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