505(b)(1)
LXPA1988
This product is a new chemical entity (NCE), specifically a kinase inhibitor targeting FLT3. Animal studies show that LXPA1988 also effectively inhibits mutated FLT3. Currently, preclinical studies and new formulation designs are underway.
Indications
FLT3-mutated Acute Myeloid Leukemia (AML)
Epidemiology
- Globally, approximately 190,000 new AML cases are reported annually.
- The number of AML patients with FLT3 mutations is around 65,000 (30-35%).
- It is classified as an orphan disease (affecting < 200,000 people)
Development strengths
- Accelerated review for market approval (authorized by EMA in December 2020 for aspacytarabine).
- Tax incentives for clinical expenses (US – 25% off).
- Post-approval market exclusivity for seven years (US) / ten years (Taiwan).
- As of 2016, the median annual cost of orphan drugs exceeded $32,000.
Lates Progress
Preclinical
IND
Phase 1
Phase 2
Phase 3
Market
Preclinical
Pre-Clinical Result
Oral administration of LXPA1988 can completely inhibit tumor growth
In the 30 mpk dosage group, all tumors in the rats regressed completely after 11 days of treatment, with no adverse reactions reported.
