Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) has had a rough time of late, just like basically everything else. However, the company recently announced something interesting: that, following constructive discussions with the Scottish Government, it will submit ORKAMBI (lumacaftor/ivacaftor) as well as SYMKEVI (tezacaftor/ivacaftor) to be used in combination with ivacaftor, to the Scottish Medicines Consortium (SMC) for appraisal.

According to the release, if accepted by the SMC for use on the NHS in Scotland, eligible patients with cystic fibrosis (CF) in Scotland could have access to these precision medicines in 2019.

Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) trumpets itself as a biotechnology company that develops medicines for serious diseases. The company focuses on developing and commercializing therapies for the treatment of cystic fibrosis (CF) and advancing its research and development programs.

It markets ORKAMBI (lumacaftor in combination with ivacaftor) to treat patients with CF 12 years of age and older who are homozygous for the F508del mutation in their cystic fibrosis transmembrane conductance regulator (CFTR) gene; KALYDECO (ivacaftor) for the treatment of patients with CF who have specific mutations in their CFTR gene, including the G551D mutation; and SYMDEKO (tezacaftor in combination with ivacaftor) to treat patients with CF 12 years of age and older who are F508del homozygous or who have 1 mutation that is responsive to tezacaftor/ivacaftor.

The company also develops VX-659 and VX-445, which are CFTR corrector compounds in Phase II clinical trial; VX-150 that is in Phase II clinical trial to treat pain; VX-128, which is in Phase I clinical trial for the treatment of pain; CTX001, an investigational gene editing treatment that is in Phase I/II trial for the treatment of beta-thalassemia and sickle cell disease; pimodivir (JNJ-63623872), which is in a Phase III clinical development program to treat influenza; and VX-210 to treat acute spinal cord injury.

It sells its products primarily to specialty pharmacy providers in North America, as well as government-owned and supported customers internationally.

The company has collaborations with Cystic Fibrosis Foundation Therapeutics Incorporated; CRISPR Therapeutics AG; Moderna Therapeutics, Inc.; BioAxone Biosciences, Inc.; Parion Sciences, Inc.; Merck KGaA; Janssen Pharmaceuticals, Inc; and Q-State Biosciences, Inc.


The Highlander?

As we discussed earlier, VRTX just announced that, following constructive discussions with the Scottish Government, it will submit ORKAMBI (lumacaftor/ivacaftor) as well as SYMKEVI (tezacaftor/ivacaftor) to be used in combination with ivacaftor, to the Scottish Medicines Consortium (SMC) for appraisal.

That comes in a context of recent action that hasn’t been particularly fun for VRTX shareholders. Over the past five days of action, shares of the stock have suffered from clear selling pressure, dropping by roughly -7%.

“Our recent conversations with the Scottish Government, and the SMC’s orphan medicines process have provided important flexibility for evaluating precision medicines, such as cystic fibrosis transmembrane conductance regulator (CFTR) modulators. Their methods reflect the innovative nature of medicines that have the potential to extend life for patients with rare diseases, like CF,” said Ludovic Fenaux, Senior Vice President, Vertex International.

“We are hopeful that, through this process, all eligible patients in Scotland could have access to our medicines soon.”

Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) generated sales of $782.8M, according to information released in the company’s most recent quarterly financial report. That adds up to a sequential quarter-over-quarter growth rate of 3.7% on the top line. In addition, the company has a strong balance sheet, with cash levels far exceeding current liabilities ($3.1B against $992.5M).

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