Marisa Wexler, MS, senior science writer —

Protego Biopharma has raised $51 million to support the development of therapies for diseases caused by protein misfolding, such as familial amyloid polyneuropathy (FAP). The series A financing was co-led by Lightspeed Venture Partners, Vida Ventures, and MPM Capital. As part of the financing, representatives from these three…

Note: This story was updated Nov. 24, 2021 to clarify that NTLA-2001 works by disrupting the TTR gene to reduce transthyretin levels.   The U.S. Food and Drug Administration (FDA) has given orphan drug designation to NTLA-2001, an investigational gene-editing therapy for familial amyloid polyneuropathy (FAP) and other forms of…

Tegsedi (inotersen), an approved treatment for familial amyloid polyneuropathy (FAP), has received a Category 1 classification by the legal body tasked with economic regulation of medications in Brazil. Category 1 is given by Câmara de Regulação do Mercado de Medicamentos to innovative treatments that are more effective than…

Onpattro (patisiran) is now reimbursed for patients with familial amyloid polyneuropathy (FAP) living across most regions of Canada, according to Alnylam Pharmaceuticals, the therapy’s developer. The announcement comes following a positive recommendation for reimbursement from the Canadian Agency for Drugs and Technologies in Health (CADTH) and the Institut…

Challenges with mental health often follow a person being told that they carry a mutation that causes familial amyloid polyneuropathy (FAP), a rare and progressive disease of adulthood, a study reports. “These results highlight the burden of [FAP] for patients and their relatives. Patients, carriers and their relatives and…

Treatment with disease-modifying therapies can extend survival in people with familial amyloid polyneuropathy (FAP), a new study indicates. The study, “Use of Drugs for ATTRv Amyloidosis in the Real World: How Therapy Is Changing Survival in a Non-Endemic Area,” was published in the journal Brain…

Alnylam Pharmaceuticals’ application for the approval of vutrisiran, its investigational therapy for familial amyloid polyneuropathy (FAP), is being reviewed by the U.S. Food and Drug Administration (FDA), with a decision expected by April 14 of next year. The agency has indicated that it is not currently planning…

Amyl Therapeutics has raised €18.3 million — about $22.2 million — to develop its ClariTY technology platform to advance therapeutic candidates for familial amyloid polyneuropathy (FAP) and other forms of amyloidosis. “The ClariTY platform offers the unique opportunity to generate therapeutic candidates able to target all amyloid fibril…

Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in Europe living with rare diseases are left behind by the start of a new decade. As part of…

Enrollment is now completed in the Phase 3 APOLLO-B trial, which is assessing the safety and efficacy of Onpattro (patisiran) to treat heart disease in people with transthyretin-mediated (ATTR) amyloidosis, a group of conditions that includes familial amyloid polyneuropathy (FAP). The study (NCT03997383) has enrolled more than…