dm+d
Unassigned
New Medicines
Welireg Von Hippel Lindau Disease-associated renal cell carcinoma
Information
Welireg
New molecular entity
Merck Sharp & Dohme (MSD)
Merck & Co (name used in US for MSD)
Development and Regulatory status
Phase II Clinical Trials
Phase II Clinical Trials
Approved (Licensed)
Aug 21Approved in US [10]
Mar 21NDA accepted for priority review by US FDA, with a PDUFA of 15/9/21 [9].
Feb 21Belzutifan for adults with von Hippel Lindau disease is awarded the first ‘ Innovation Passport ’ by the MHRA, NICE and the SMC. This means patients could benefit much sooner from this treatment and it will be accelerated through the approval process; the Innovative Licensing and Access Pathway (ILAP). By incorporating patient views, both on the benefits and risks of medicines in the pathway, and on how to improve patient outcomes throughout the product lifecycle, medicines can be developed to meet patient requirements more successfully [8].
Jul 20FDA grant Breakthrough Therapy orphan drug designation to MK-6482 (formerly PT2977) for VHL disease [6].
May 19Merck (MSD) has announced acquisition of Peloton Therapeutics [5].
Category
Potent, selective HIF-2α inhibitor. Selective inhibition of HIF-2α transcription factor leads to impaired hypoxic signalling in cancer cells and blocks transcription of several genes involved in oncogenesis [1,2]
The incidence of VHL disease is about 1 per 36,000 live births. Approx 60% of all patients with VHL will develop RCC or renal cysts. Both are typically bilateral and found in multiple locations within the kidney. RCCs are found in 24% to 45% of patients with VHL and are the primary inherited renal cancer. The mean age of RCC onset is 39 years old, which is 25 years less than the mean age of the typical patient [3].
Von Hippel Lindau Disease-associated renal cell carcinoma
Oral
Further information
Yes
Trial or other data
Aug 21PII trial (NCT03401788) is now due to complete collection of primary outcome data in Mar 26 [11].
Nov 20PII trial (NCT03401788) is now due to complete collection of primary outcome data in Mar 22 [7].
Apr 19Peloton announce completion of patient enrolment in PII trial (NCT03401788) [1,2].
Mar 18Open-label PII trial (NCT03401788) to investigate safety and efficacy of PT 2977 in patients with Von Hippel Lindau disease with at least one measurable renal cell carcinoma tumour started. The trial plans to enrol 50 subjects in the US, Denmark, France and UK, and primary completion date in Mar 2020 [4].
Evidence based evaluations
Welireg Advanced clear-cell renal cell carcinoma - second-line
Information
Welireg
Licence extension / variation
Merck Sharp & Dohme (MSD)
Merck & Co (name used in US for MSD)
Development and Regulatory status
Phase III Clinical Trials
Phase III Clinical Trials
Phase III Clinical Trials
Category
Potent, selective HIF-2α inhibitor. Selective inhibition of HIF-2α transcription factor leads to impaired hypoxic signalling in cancer cells and blocks transcription of several genes involved in oncogenesis [1,2]
RCC affects ~4 in 10,000 people in the EU, equivalent to a total of ~203,000 people
Advanced clear-cell renal cell carcinoma - second-line
Oral