Products

Date: 2016-06-30

Type of information: Granting of the orphan status in the US

Product name: TK216 - small molecule Ewing Sarcoma breakpoint region 1/Friend leukemia virus integration 1 fusion protein inhibitor

Compound: small molecule Ewing Sarcoma breakpoint region 1/Friend leukemia virus integration 1 fusion protein inhibitor

Therapeutic area: Cancer - Oncology - Rare diseases

Action mechanism: TK216 is a first-in-class small molecule that inhibits the biological activity of ets-family transcription factor oncoproteins in a variety of tumor types, stopping cancer cell growth and tumor formation. In Ewing sarcoma, it is designed to target a single and well-characterized genetic mutation that causes the disease. TK216 is being developed collaboratively by Georgetown University and Oncternal.

Company: Tokalas (USA - CA) now Oncternal Therapeutics (USA - CA)

Disease: Ewing sarcoma

Latest news:

• • On June 30, 2016, the FDA has granted orphan drug designation for small molecule Ewing Sarcoma breakpoint region 1/Friend leukemia virus integration 1 fusion protein inhibitor for treatment of Ewing sarcoma.
• On June 20, 2016, Oncternal Therapeutics announced that TK216 has received Fast Track Designation from the FDA for the treatment of patients with Ewing sarcoma who have relapsed or are refractory to standard of care therapy. Oncternal is in the process of initiating a first-in-human Phase 1 trial in relapsed or refractory Ewing sarcoma.

Patents:

Submission of marketing authorization application USA :

Submission of marketing authorization application UE:

Withdrawal of marketing authorization application USA:

Withdrawal of marketing authorization application UE:

US authorization:

UE authorization:

Favourable opinion UE:

Favourable opinion USA:

Orphan status USA: 2016-06-30

Orphan status UE:

Pediatric exclusivit _USA:

Pediatric exclusivity UE:

OTC status:

Other news:

Is general: Yes