July/August 2021 • PharmaTimes Magazine • 8-9
// MEDICINE //
PD-1 inhibitor Libtayo cleared for basal cell carcinoma
Sanofi and Regeneron’s PD-1 inhibitor Libtayo (cemiplimab) has won a green light in Europe as the first immunotherapy to treat adults with locally advanced or metastatic basal cell carcinoma (BCC).
Specifically, the drug has been approved to treat patients who have progressed on or are intolerant to a hedgehog pathway inhibitor (HHI).
BCC is the most common type of skin cancer worldwide, representing up to 80% of non-melanoma skin cancers, and incidence is increasing across many European countries. The vast majority of BCCs are caught early and easily cured with surgery and/or radiation, but a small proportion of cases can develop into advanced forms of the disease, penetrating deeper into surrounding tissues or spreading to other parts of the body, thus becoming more difficult to treat.
Libtayo's approval in BCC follows data showing an objective response rate (ORR) of 32% by independent central review and 29% by investigator assessment. Also, around 90% of patients across both groups had a duration of response (DOR) of six months or longer, and the median DOR has not been reached for either group.
“Libtayo is the first immunotherapy to show a clinical benefit in patients with advanced BCC after HHI therapy in a pivotal trial, and with this first-in-class approval has the potential to transform treatment for patients in Europe whose cancer has progressed despite HHI treatment,” said Israel Lowy, senior vice president, Translational and Clinical Sciences, Oncology at Regeneron.
Libtayo is now approved for three advanced cancers in the EU: BCC, non-small cell lung cancer (NSCLC) and cutaneous squamous cell carcinoma (CSCC).
Early access scheme for new Pompe disease drug
Patients with late-stage Pompe disease who are failing to respond to enzyme replacement therapy will now have early access to an alternative treatment option, after Amicus Therapeutics' cipaglucosidase alfa with miglustat was approved for the UK's Early Access to Medicine Scheme (EAMS).
The Medicines and Healthcare Regulatory products Agency (MHRA) has issued a positive scientific opinion for use of the treatment in adults with late-onset Pompe disease previously treated with alglucosidase alfa, based on current data.
This means that doctors will be able to prescribe the treatment to eligible patients with the rare, inherited disorder while its licensing is being considered by regulators.
As such, under EAMS the risk and legal responsibility for prescribing remains with the physician, and the opinion and EAMS documentation published by the MHRA are intended only to inform decision-making and not to recommend use.
CHMP nod for BMS' Abecma
Bristol Myers Squibb’s (BMS) CAR T-cell therapy Abecma (idecabtagene vicleucel) has won backing from the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) for the treatment of relapsed and refractory multiple myeloma.
Abecma is a B-cell maturation antigen (BCMA)-directed CAR T-cell immunotherapy, designed to treat relapsed and refractory multiple myeloma patients who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody.
The decision rides on results from the Phase II KarMMa study, which evaluated Abecma in 128 patients with heavily pretreated and highly refractory multiple myeloma, and found an overall response rate (ORR) of 72%, with 28% of patients achieving a stringent complete response (sCR).
In addition, a median time to response of 30 days was observed, as well as a median duration of response of 11 months for all responders and 19 months for those who achieved sCR, and of the patients who achieved sCR, an estimated 65% had remission lasting at least 12 months.
“As the first CAR T-cell therapy for relapsed and refractory multiple myeloma to receive a positive CHMP opinion, Abecma represents a potential new treatment approach for patients in Europe battling this incurable blood cancer,” said Noah Berkowitz, senior vice president, cellular therapy development, BMS
MHRA approves Venclyxto for newly diagnosed AML
The UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) has approved AbbVie and Roche’s Venclyxto (venetoclax) for the treatment of newly diagnosed acute myeloid leukaemia (AML).
The decision allows doctors to prescribe the drug in combination with a hypomethylating agent for patients who are ineligible for intensive chemotherapy, thus offering an alternative treatment option in this setting.
The approval is based on results from the Phase III VIALE-A study as well as the Phase Ib M14-358 clinical trial. In VIALE-A, patients who received Venclyxto in combination with azacitidine showed a statistically significant greater median overall survival (OS) compared to patients receiving azacitidine alone.
Meanwhile, the M14-358 study, which evaluated Venclyxto in combination with hypomethylating agents, demonstrated an overall safety profile that was consistent with the known safety profiles of the drug combined with azacitidine or the two medications alone.
“AML is an incredibly aggressive form of blood cancer, and patients who are diagnosed with this disease often cannot tolerate intensive chemotherapy due to advanced age and coexisting conditions” said Belinda Byrne, medical director, AbbVie UK. “Regulatory approval of Venclyxto is an important step forward in raising the standard of care for these patients, offering the potential to achieve remission with a manageable safety profile,” she added.
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