July / August 2020 • PharmaTimes Magazine • 8-9
// MEDICINE //
As PharmaTimes was going to press reports emerged that Gilead's remdesivir had been issued a one-year conditional approval in Europe, becoming the first medicine cleared to treat COVID-19 after winning a positive recommendation from the EMA’s human medicines committee (CHMP).
If approved, doctors will be able to prescribe the drug – which will be on the market under the brand name Veklury – for the treatment of COVID-19 in patients aged 12 years and over with pneumonia who require supplemental oxygen.
Data on the drug was assessed in “an exceptionally short time frame” through a rolling review procedure, an approach used by EMA during public health emergencies to assess data as it becomes available.
From April 30, the CHMP began assessing data on quality and manufacturing, non-clinical and preliminary clinical data and safety data from compassionate use programmes, well in advance of the submission of the marketing application on June 5.
The decision to back the drug's approval in this setting is mainly based on findings of the US National Institute of Allergy and Infectious Diseases (NIAID) study NIAID-ACTT-11, which found that patients treated with remdesivir recovered after about 11 days, compared with 15 days for patients given placebo.
However, the agency stressed that this effect was not seen in patients with mild to moderate disease: time to recovery was five days for both the remdesivir group and the placebo group. For patients with severe disease, who constituted approximately 90% of the study population, time to recovery was 12 days in the remdesivir group and 18 days in the placebo group.
Taking into consideration the available data, the CHMP felt the balance of benefits and risks had been shown to be positive in patients with pneumonia requiring supplemental oxygen; ie, the patients with severe disease.
As the recommended authorisation is a conditional one, Gilead would have to submit final reports of the remdesivir studies by December 2020, and further data on the quality of the medicine, as well as the final data on mortality, by August 2020 in order to better characterise the drug's effectiveness and safety.
Vertex has reached an agreement with NHS England to reimburse its triple combination therapy Kaftrio (ivacaftor/tezacaftor/elexacaftor) in combination with Kalydeco (ivacaftor), should the medicine win European approval.
The new expanded agreement includes reimbursed access to Vertex’ currently licensed medicines – Kalydeco (ivacaftor), Orkambi (lumacaftor/ivacaftor) and Symkevi (tezacaftor/ivacaftor), as well as the triple combination therapy if approved – and any future additional licensed indications for all of these medicines.
The move follows recent backing for Kaftrio in combination with Kalydeco from the European Medicines Agency’s human medicines committee (CHMP), which issued a green light for its use to treat people with CF ages 12 and older with one F508del mutation and one minimal function mutation (F/MF) or two F508del mutations (F/F) in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
As part of the deal with NHS England, Vertex has committed to submit Orkambi, Symkevi and Kaftrio to the National Institute for Health and Care Excellence (NICE) within an agreed timetable, allowing for a period of real-world data collection on the medicines, it said.
If approved, three in five patients with the deadly genetic condition could benefit from the ‘triple combination’ treatment, and nine in ten CF patients – more than 7,000 people – will now benefit from a therapy which tackles the underlying causes of the disease.
“I’m pleased that NHS England has recognised the value of Kaftrio, and that Vertex and NHS England have been able to work quickly, collaboratively and flexibly to expand the existing reimbursement agreement to include the triple combination therapy in advance of the medicine being licensed,” said Vertex' chief executive and president Reshma Kewalramani.
“This will ensure that eligible patients in England will be among the first in Europe to benefit from access to this innovative medicine upon approval.”
Commercial terms of the agreement are confidential, but NHS England noted that the approach achieved is consistent with its other managed access agreements reached with the pharmaceutical industry.
NICE will not be recommending Janssen's Zytiga (abiraterone acetate) for treating newly diagnosed high-risk metastatic hormone-sensitive prostate cancer (mHSPC).
Around 4,000 men newly diagnosed with the condition would have been eligible for treatment with Zytiga in combination with prednisone/prednisolone and ADT, had the therapy won NICE backing for NHS funding.
Currently, men first diagnosed with advanced prostate cancer are given hormone therapy/docetaxel chemotherapy, or hormone therapy alone, but around three-quarters are unable to tolerate it or choose not to take it because of potentially serious side effects.
NICE's rejection of NHS funds for the drug in England and Wales highlights the postcode lottery of care that exists across the UK, as the Scottish Medicines Consortium (SMC) has approved use of the drug in the first-line setting, based on the same evidence base.
The Institute of Cancer Research (ICR) has expressed disappointment with the decision, and is now calling for NHS England and Janssen to come to an agreement on pricing.
The STAMPEDE and LATITUDE trials found that first-line Zytiga “extended the time patients lived without their disease coming back, halved subsequent problems like bone fracture and the need for radiotherapy and offered them a better quality of life compared with a combination of hormone therapy and docetaxel chemotherapy," it said.
“Abiraterone offers men a clear improvement in quality of life compared with chemotherapy or hormone therapy alone,” added Professor Nick James, Professor of Prostate and Bladder Cancer Research at The ICR. “The quality of life benefits of abiraterone are particularly important for older men who might not be well enough to receive chemotherapy. It is especially frustrating that there will be such stark differences across the UK, with men in Scotland able to access abiraterone first line, but those in England and Wales facing the prospect of missing out.”
"We are currently reviewing the FAD in detail and we will assess our options for a potential challenge to this decision, particularly given the evidence supporting the value of abiraterone in patients who are ineligible for chemotherapy," said Sarah Scanlon, Janssen's business unit director Oncology.