BUSINESS corner Akcea receives their first drug approval from the European Commission Ottawa-based Akcea Therapeutics Inc. receives their first drug approval from the European Commission for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR). Tegsedi is now the world’s first and only RNA-targeted therapeutic approved for patients with hATTR. “With the EC’s decision, Tegsedi is now the world’s first and only RNA-targeted therapeutic approved for patients with hATTR amyloidosis,” says Paula Soteropoulos, chief executive officer at Akcea Therapeutics. “With subcutaneous delivery, Tegsedi puts treatment in the patients’ hands while bringing the significant benefits shown in the NEURO-TTR study in both measures of neuropathy and quality of life for people living with this serious and fatal disease. This is an important day for the hATTR amyloidosis community as we believe Tegsedi enables people and their families impacted by this disease to move forward with their lives. Today is a milestone for Akcea with our first drug approval. It is an achievement we share with the courageous hATTR patient community in Europe and around the globe.
We are ready to launch Tegsedi along with our patient and physician support services across Europe,” she adds. The abnormal formation and aggregation of transthyretin (TTR) protein results in TTR amyloid deposits throughout the body and is the underlying cause of hATTR amyloidosis. Tegsedi is designed to block the production of the TTR protein. In the NEURO-TTR study, treatment with Tegsedi produced substantial reductions in the levels of the TTR protein regardless of mutation type or stage of the disease. The European Commission’s approval of Tegsedi was based on results from the Phase 3 NEURO-TTR study in patients with hATTR amyloidosis with symptoms of polyneuropathy. The study demonstrated that patients treated with Tegsedi experienced significant benefit compared to patients treated with a placebo across both co-primary endpoints: the Norfolk Quality of Life QuestionnaireDiabetic Neuropathy (Norfolk QoL-DN) and modified Neuropathy Impairment Score +7 (mNIS+7), a measure of neuropathic disease progression. “Today, we are thrilled to see our success-
ful research and development efforts result in the approval of an important new medicine for patients with hATTR amyloidosis. Using our antisense technology platform, we set out to design a therapy to block the production of the underlying cause of this disease, the TTR protein,” says Brett P. Monia, Ph.D., chief operating officer at Ionis Pharmaceuticals, an affiliate of Akcea Therapeutics. “Approval of Tegsedi further establishes Ionis as a multi-product company. We are confident that the experienced team at Akcea will deliver on the promise of Tegsedi. We are grateful to all of the physicians and patients who participated in the Tegsedi clinical program and who made this landmark approval possible,” he adds. Health Canada granted priority review for the New Drug Submission (NDS) filed for Tegsedi and is anticipating approval at some point this year. To see this story online visit https://biotechnologyfocus.ca/akceareceives-their-first-drug-approval-fromthe-european-commission/
PlantForm collaborates to manufacture biosimilar to the South African market Guelph-based PlantForm Corporation of Canada is stretching across the ocean to collaborate with South Africa’s Council for Scientific and Industrial Research (CSIR) and BGM Pharmaceuticals to produce a biosimilar of trastuzumab using PlantForm’s vivoXPRESS® manufacturing system. This agreement will permit the CSIR to produce a biosimilar monoclonal antibody (trastuzumab) using the tobacco plantbased vivoXPRESS® expression technology on a laboratory scale. The CSIR will demonstrate the utility of PlantForm’s proprietary technology to produce a trastuzumab drug candidate with the long-term goal of manufacturing trastuzumab, and other possible drug candidates, in South Africa for sale in African markets. “This agreement represents a significant opportunity for PlantForm,” says Dr. Don Stewart, PlantForm’s president and CEO. “The South African pharmaceutical sector is the largest in the region, and by working with the CSIR and BGM Pharmaceuticals we continue to advance our global strategy for biosimilar drug development that will make life-saving medications more widely available at much lower cost.” 8 BIOTECHNOLOGY FOCUS August/September 2018
(Photo Credit: PlantForm Corp.) The vivoXPRESS® system is revolutionary, using fast-growing tobacco plants to produce a wide range of biologic (large molecule) drugs, including monoclonal antibodies and therapeutic proteins, at a fraction of the cost of manufacturing the same drugs using conventional methods. Studies have shown that PlantForm’s plant-produced trastuzumab drug candidate is equally as effective at inhibiting the growth of HER2positive breast cancer cells as the commercial brand-name antibody Herceptin. “We’re pleased to be working with PlantForm and BGM Pharmaceuticals to further development of the vivoXPRESS® technology,” says Dr. Tsepo Tsekoa, who heads the CSIR’s biomanufacturing technology demonstration research group. “The CSIR is committed to utilizing and enhancing our capability in
biologics production to assist South African industry with development and localization of cost-effective technology solutions for biopharmaceutical manufacture. This collaboration will strengthen this commitment and help us expand our research into using plant-based processes to develop biologics.” Looking farther into the future, the collaboration will advance BGM Pharmaceuticals’ mission to develop new biopharmaceuticals and improve existing therapies for emerging African markets, while at the same time showcasing some of Canada’s innovative and forwardthinking technologies. “We’re very excited about the opportunity to bring PlantForm’s low-cost manufacturing platform to the South African market to help make life-saving biopharmaceuticals more affordable and more widely available throughout the region,” says Martin Magwaza, BGM’s president and CEO. To see this story online visit https://laboratoryfocus.ca/plantformcollaborates-to-manufacture-biosimilar-to-the-south-african-market/
