Mitazalimab gains EMA orphan drug designation for pancreatic cancer

31st August 2023

Mitazalimab has received orphan drug designation status by the European Medicines Agency (EMA) for the treatment of patients with pancreatic cancer, its manufacturer Alligator Bioscience has announced.

A human CD40 agonistic antibody targeting CD40, mitazalimab kickstarts the cancer-immunity cycle by priming and activating tumour-specific T cells. Targeting CD40 with mitazalimab has the potential to augment responses to chemotherapy.

Pancreatic ductal adenocarcinoma (PDAC) is known to be the fourth-leading cause of cancer related mortality in the world and has a poor prognosis, with a five-year survival rate of below 5%.

To qualify for the EMA’s orphan designation, a medicine must be intended for the treatment, prevention or diagnosis of rare, life-threatening or chronically debilitating diseases that affect fewer than five in 10,000 persons in the EU. Medicines that meet these criteria are eligible for financial and regulatory incentives that include 10 years of marketing exclusivity in the EU after product approval.

The EMA orphan drug designation follows a similar approval by the FDA in May 2023.

Commenting on the EMA approval for orphan drug designation status, Søren Bregenholt, CEO of Alligator Bioscience, said: ‘We are very pleased that the European Medicines Agency has granted orphan designation to our lead asset mitazalimab in the treatment of pancreatic cancer.

‘It is our second orphan designation this year following the FDA‘s decision to grant us [orphan drug designation] in May, meaning mitazalimab now has stronger commercial protection through market exclusivity in these two key markets. This latest designation adds to the momentum we are building in our efforts to bring this promising drug candidate to market.‘

Mitazalimab and OPTIMIZE-1

Mitazalimab is currently being evaluated in the phase 1b/2 OPTIMIZE-1 trial in combination with mFOLFIRINOX chemotherapy for adult patients with previously untreated metastatic PDAC.

In the trial, participants receive mitazalimab and mFOLFIRINOX via intravenous infusions following a 14-day cycle schedule. Mitazalimab is administered two days after mFOLFIRINOX, except for the first cycle of 21 days, where the drug is administered on days one and 10 with infusion of mFOLFIRINOX starting on day eight.

Interim results from OPTIMIZE-1 released in June 2023, showed a deepening of tumour response and an increase in the objective response rate (ORR) from 52% to 57% in a cohort of 23 patients.

In the full study cohort of 57 patients, there was an interim ORR of 44%, and this is expected to further improve with longer follow-up. A median duration of response of 8.7 months was also reported.

Decongestant medicines under review by regulators after rare side effects

27th February 2023

Decongestant drugs such as pseudoephedrine are under review by health regulators following concerns over rare but serious side effects

The Medicines and Healthcare Products Regulatory Agency said it had initiated a review after regulators in France alerted the European Medicines Agency (EMA) about a small number of cases.

EMA officials first announced they were looking into the issue on 10 February 2023 following concerns about posterior reversible encephalopathy syndrome (PRES) and reversible cerebral vasoconstriction syndrome (RCVS) related to use of the decongestant medicines.

Both conditions can lead to reduced blood supply to the brain and may cause major and life-threatening complications, including seizures, the EMA said.

There had been a small number of reported cases of PRES and RCVS associated with pseudoephedrine-containing medicines and a committee would look at the evidence to decide whether the marketing authorisation in the EU should be maintained, varied, suspended or withdrawn.

The MHRA stressed that the side effects that had been reported with use of the medicines were extremely rare.

The products, which include Sudafed, Actifed and Neurofen Cold and Flu, already include warnings about the rare side effects as well as more common ones such as headache and dizziness. 

Officials said they had received two Yellow Card reports on this issue including one case of PRES where the person recovered and one for RCVS where the outcome was reported as unknown.

A spokesman said: ‘We keep the safety of all medicines under close review to ensure that the benefits outweigh any risks – the safety of the public is our top priority.

‘We are reviewing the available evidence regarding the use of medicines containing pseudoephedrine and the risk of posterior reversible encephalopathy syndrome (PRES) and reversible cerebral vasoconstriction syndrome (RCVS), which have been very rarely reported with these medicines. We will provide any further advice as appropriate. 

‘We would also like to remind patients and parents/carers to report any suspected side effects to our Yellow Card scheme.

‘If you have any concerns about your medicine, please seek advice from a healthcare professional.’

This article first appeared in our sister publication Pulse