Products

Date: 2016-11-21

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

Product name: PQR309 - 5-(4,6-dimorpholino-1,3,5-triazin-2-yl)-4-(trifluoromethyl)-pyridin-2-amine

Compound: 5-(4,6-dimorpholino-1,3,5-triazin-2-yl)-4-(trifluoromethyl)-pyridin-2-amine

Therapeutic area: Cancer - Oncology - Rare diseases

Action mechanism: phosphoinositide 3-kinase (PI3K) inhibitor/mTOR inhibitor. PQR309 is an orally bioavailable pan inhibitor of phosphoinositide-3-kinases (PI3K) and inhibitor of the mammalian target of rapamycin (mTOR), with potential antineoplastic activity. PQR309 inhibits the PI3K kinase isoforms alpha, beta, gamma and delta and, to a lesser extent, mTOR kinase, which may result in tumor cell apoptosis and growth inhibition in cells overexpressing PI3K/mTOR. Activation of the PI3K/mTOR pathway promotes cell growth, survival, and resistance to both chemotherapy and radiotherapy. As mTOR, a serine/threonine kinase downstream of PI3K, may also be activated independent of PI3K, this agent may potentially be more potent than an agent that inhibits either PI3K kinase or mTOR kinase. By inhibiting mTOR to a lesser extent than PI3K, PQR309 does not interfere with the mTOR-mediated negative feedback loop on PI3K signaling. Blocking the negative feedback loop would potentially increase PI3K signaling and decrease therapeutic efficacy.

Company: PIQUR Therapeutics (Switzerland)

Disease: primary central nervous system lymphoma (PCNSL)

Latest news:

• • On December 2, 2016, Piqur Therapeutics announced that the FDA has granted orphan drug designation to PIQUR’s lead compound PQR309 (5-(4,6-dimorpholino-1,3,5-triazin-2-yl)-4-(trifluoromethyl)-pyridin-2-amine) for the treatment of primary central nervous system lymphoma (PCNSL). PCNSL is a very rare and aggressive form of lymphoma involving brain and its linings, eyes or spinal cord. The disease affects less than 1 person in 100,000 in the USA with approximately 7,500 new cases reported annually in the USA, Europe and Japan. Treatment options available for PCNSL are limited, and the prognosis with current therapies is poor.

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: 2016-11-21

Orphan status UE:

Pediatric exclusivit _USA:

Pediatric exclusivity UE:

OTC status:

Other news:

Is general: Yes