Like PYC’s lead asset, VP-001 for the treatment of Retinitis Pigmentosa 11, VP-002, for the treatment of Autosomal Dominant Optic Atrophy caused by mutations in the OPA1 gene, is a rare disease drug.
Though the patient populations for rare diseases can be quite small, potential regulatory as well as economic benefits make rare diseases (also called orphan diseases) attractive for drug development.
Orphan Drug Designation
The United States, EU and Japan have all implemented programs that provide incentives to orphan drug developers.
For example, in the United States, orphan designated drugs (those addressing conditions affecting <200,000 in the US) are eligible for:
Market exclusivity for 7 years post approval (the EU provides 10 yrs)
Tax credits of 50% off the clinical drug testing cost, awarded upon approval
Eligibility for waiver of NDA/BLA application fee (approx. $3 million value)
OOPD (FDA Office of Orphan Products Development) assistance during the development process
Fast Track
Fast track is designed to facilitate the development, and expedite the review of drugs that treat serious conditions and fill an unmet medical need.
Fast Track designation provides eligibility for some or all of the following:
More frequent meetings with FDA to discuss the drug's development plan and ensure collection of appropriate data needed to support drug approval.
More frequent written communication from FDA on matters such as proposed clinical trial design.
Eligibility for Accelerated Approval and Priority Review, if relevant criteria are met.
Rolling Review, which means that a drug company can submit completed sections of its NDA or BLA for review by the FDA, rather than waiting until every section of the NDA is completed before the entire application can be reviewed.
Priority Review Designation
A Priority Review designation means the FDA aims to complete marketing approval drug review within 6 months, compared to 10 months under standard review.
Breakthrough Therapy
Breakthrough Therapy designation is a process designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s).
Breakthrough Therapy designation provides eligibility for the following:
All Fast Track designation features.
Intensive guidance on an efficient drug development program, beginning as early as Phase 1.
There are strong competitive headwinds in the orphan drug space. Big pharma is typically discouraged from spending huge amounts of resources on what it considers to be a small group of patients, a minimum market.
Patient Advocacy Support
Rare disease often comes with strongly supportive patient advocate communities. Potential areas of benefit include patient recruitment, funding, lobbying of regulatory authorities and government and speed of market penetration.
Higher Probability of Success
The average non-oncology Phase 1 drug has ~ 10% chance of eventual FDA approval. Orphan genetic drugs, such as those being developed by PYC, have ~45% chance of reaching market from Phase 1.
Faster and Lower Cost Path to Market
40% reduction in clinical development timeline compared with non-orphan drugs, with opportunity to combine phases 2 and 3 and obtain accelerated approval.
40% lower cost of clinical trials with smaller patient numbers required.
Higher Pricing
Median price differential (orphan/non-orphan) in 2018 for top 100 drugs was 7.0.
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