WHAT IS orphan disease designation?

The Orphan Drug Act (ODA) provides for granting special status to a drug or biological product (“drug”) to treat a rare disease or condition upon request of a sponsor. This status is referred to as orphan designation (or sometimes “orphan status”).

How do you qualify for orphan drug designation?

Criteria for Orphan Drug Designation

The product must be intended for use in a rare disease or condition.
Adequate documentation or prevalence data must demonstrate that the intended condition is rare.

How much is an orphan drug designation worth?

A Rise in Orphan Drug Designation (ODD) Approvals The increase in approvals is leading industry experts to value the global orphan drug market at $300 billion by 2026, more than 20% of global prescription drug sales3, up from $132 billion in 2019.

What does orphan status of a drug mean?

A status given to certain drugs called orphan drugs, which show promise in the treatment, prevention, or diagnosis of orphan diseases. An orphan disease is a rare disease or condition that affects fewer than 200,000 people in the United States. Orphan diseases are often serious or life threatening.

What is the benefit of orphan drug designation?

The purpose of the designation was to create financial incentives for companies to develop new drugs and biologics for rare diseases. These incentives include a partial tax credit for clinical trial expenditures, waived user fees, and eligibility for 7 years of marketing exclusivity [1].

How long does orphan drug designation last?

In the world of exclusivity, there is one overarching rule: all good things must come to an end. New chemical entity (NCE) exclusivity expires after five years (sometimes four); orphan drug exclusivity ends after seven years; and even Reference Product exclusivity expires eventually – after a whopping 12 years.

What is the difference between orphan drug designation and approval?

The orphan designation is part of the approval process Submitting an orphan designation request is unrelated to the drug approval process. In fact, the orphan application can be filed anytime in the drug development process before NDA/BLA submission, even prior to IND filing.

Can you lose orphan drug designation?

Any benefits attendant to designation (such as orphan-exclusive approval) will cease once designation is voluntarily withdrawn, from the date of FDA’s acknowledgement letter. If a sponsor voluntarily withdraws designation, FDA will publicize such withdrawal in accordance with § 316.28.

What is world’s rarest disease?

1. RPI deficiency. According to the Journal of Molecular Medicine, Ribose-5 phosphate isomerase deficiency, or RPI Deficinecy, is the rarest disease in the world with MRI and DNA analysis providing only one case in history.

How long does orphan designation last?

What makes a drug qualify for orphan status?

What was the Orr for dabrafenib in NSCLC patients?

The ORR for patients who received single-agent dabrafenib was 27% (95% CI: 18%, 38%) and the median DoR was 9.9 months. The incidence and severity of adverse reactions occurring in patients with NSCLC were generally similar to those reported in prior approvals for patients with melanoma.

When did dabrafenib get an orphan drug designation?

Orphan Drug Designation was granted in 2014 to dabrafenib as a single agent for the treatment of patients with BRAF mutation positive NSLC and in 2015 combination with trametinib. A description of FDA expedited programs is available in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics.

How to contact the FDA orphan drug program?

When transmitting information to the Orphan Drug Designation Program via email, please utilize the mailbox [email protected]. The use of automated read receipt is recommended to avoid the need to call to verify receipt of the email.