Products

Date: 2016-01-15

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

Product name: LOXO- 101 (larotrectinib - (S)-N-(5-((R)-2-(2,5-difluorophenyl)pyrrolidin-1-yl)pyrazolo[1,5-a]pyrimidin-3-yl)-3-hydroxypyrrolidine-1-carboxamide hydrogen sulfate

Compound: (S)-N-(5-((R)-2-(2,5-difluorophenyl)pyrrolidin-1-yl)pyrazolo[1,5-a]pyrimidin-3-yl)-3-hydroxypyrrolidine-1-carboxamide hydrogen sulfate

Therapeutic area: Cancer - Oncology - Rare diseases

Action mechanism:

• enzyme inhibitor/kinase inhibitor. LOXO-101 is a potent, oral, selective inhibitor of tropomyosin receptor kinase (TRK) signaling molecules. The TRK family (TRKA, TRKB, and TRKC) has been implicated in diverse tumor types such as lung cancer, head and neck cancer, melanoma, colorectal cancer, sarcoma, and breast cancer. LOXO-101 was built specifically to inhibit TRK and is currently being evaluated in a Phase 1 dose escalation trial for patients with advanced solid tumors.

Company: Loxo Oncology (USA - CT)

Disease: soft tissue sarcoma

Latest news:

• • On January 15, 2016, Loxo Oncology announced that the European Medicines Agency (EMA) has granted the company orphan drug designation for LOXO-101 for treatment of patients with soft tissue sarcoma. In an ongoing Phase 1 clinical trial, LOXO-101 has demonstrated encouraging preliminary efficacy. LOXO-101 is also being evaluated in a global Phase 2 multi-center basket trial in patients with solid tumors that harbor TRK gene fusions.
• • On December 8-10, 2015, the Committee for Orphan Medicinal Products (COMP) adopted a positive opinion recommending (S)-N-(5-((R)-2-(2,5-difluorophenyl)pyrrolidin-1-yl)pyrazolo[1,5-a]pyrimidin-3-yl)-3-hydroxypyrrolidine-1-carboxamide hydrogen sulfate for treatment of soft tissue sarcoma,for designation as orphan medicinal product.
• • On August 31, 2015, the FDA has granted orphan drug designation for (S)-N-(5-((R)-2-(2,5-difluorophenyl)pyrrolidin-1-yl)pyrazolo[1,5-a]pyrimidin-3-yl)-3-hydroxypyrrolidine-1-carboxamide hydrogen sulfate for the treatment of soft tissue sarcoma. LOXO-101 is currently being evaluated in a Phase 1 dose escalation trial for patients with advanced solid tumors (NCT02122913).

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: 2015-08-31

Orphan status UE: 2016-01-15

Pediatric exclusivit _USA:

Pediatric exclusivity UE:

OTC status:

Other news:

Is general: Yes