The decision by the Therapeutic Goods Administration (TGA) means that myeloma patients in Australia will be the first to have access to this medication.
Specifically, Aplidin was approved for patients who relapsed after three lines of treatment, including proteasome inhibitors or immunomodulators. The therapy also can be given as third-line treatment to patients who are refractory or intolerant to proteasome inhibitors or immunomodulators.
Aplidin is extracted from the sea squirt (Aplidium albicans). It binds to the protein product of the EEF1A2 gene, believed to play an important role in cancer development. Aplidin leads to tumor cell death via apoptosis, which refers to “programmed” cell death, as opposed to cell death caused by injury.
“Aplidin provides a chance for some myeloma patients to extend their lives,” Andrew Spencer, the head of the Malignant Haematology and Stem Cell Transplantation Service at Alfred Hospital, in Melbourne, said in a press release. Spencer also noted the importance of this approval, as patients not responding to prior therapies have “very limited treatment options.”
Jeff Szer, the Australian principal investigator on the pivotal Phase 3 trial of Aplidin, known as ADMYRE (NCT01102426), cited the good efficacy and tolerability results of a study that enrolled more Australian patients “than anywhere else in the world.” These patients, he added, “paved the way for others to have access to a new and novel therapy.”
Results of ADMYRE showed that combining Aplidin with dexamethasone significantly extended survival compared to dexamethasone alone in patients who had received three to six prior therapies.
“This really means that some patients with advanced myeloma have the possibility of improved outcomes,” said Szer, who is a hematologist at the Peter MacCallum Cancer Centre and Royal Melbourne Hospital.
Singapore-based Specialised Therapeutics Asia, which licensed Aplidin from Madrid-based PharmaMar, will continue providing Aplidin to eligible Australian patients at no cost through a Compassionate Access Program, before national reimbursement. The company holds exclusive rights to market and distribute Aplidin in Australia, Singapore, and 12 other Southeast Asian countries.
“It is not often that Australian patients are the first in the world to access new medicines,” said Carlo Montagner CEO of Specialised. “In this case, the TGA is at the forefront, with decision-makers recognising the great need that exists in multiple myeloma.”
Jose Maria Fernandez Sousa-Faro, PharmaMar’s president, said the approval corroborates the work conducted at the company. “Patients and the medical community will now have a new therapeutic alternative with a new mechanism of action, that is different from the products currently in use,” he said.
Luis Mora, managing director of PharmaMar’s Oncology Business Unit, said that Aplidin’s approval “is a very important step forward for the company.” He also noted that “together with our partners, we are initiating procedures for other markets, such as South America, Mexico, Canada, Asia and Israel.”
In December 2017, the Committee for Medicinal Products for Human Use, an arm of the European Medicines Agency, recommended against the approval of Aplidin for the same patient population. The decision is being appealed to the Luxembourg Court.
Aplidin was granted orphan drug status in Switzerland, the U.S. and Europe.