PTC Therapeutics Announces Waylivra Approval in Brazil as First Treatment for Familial Chylomicronemia Syndrome

PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the Brazilian Health Regulatory Agency, ANVISA (Agência Nacional de Vigilância Sanitária), has approved Waylivra™ (volanesorsen) as the first treatment for familial chylomicronemia syndrome (FCS) in Brazil. FCS is a rare genetic disease which results in significant disease burden to patients including potentially fatal pancreatitis and chronic complications due to permanent organ damage.

"We are excited to announce Waylivra's approval in Brazil as the first treatment for the underlying cause of FCS," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics. "This approval is another example of PTC leveraging its strong capabilities in Latin America to bring transformational therapies to patients with high unmet needs."

ANVISA's approval of Waylivra is based on results from the Phase 3 APPROACH study and the APPROACH Open-Label Extension study and is supported by results from the Phase 3 COMPASS study. Results from the Phase 3 APPROACH trial, the largest study ever conducted in patients with FCS, show that treatment with Waylivra reduced triglycerides 94% when compared to placebo. All patients in the trial maintained a low-fat diet.

Waylivra is a self-administered, subcutaneous injection that comes in a single-use, prefilled syringe and is the first approved pharmacotherapy indicated for the treatment of FCS. It is an innovative antisense oligonucleotide (ASO), with a novel mode of action designed to reduce the production of apolipoprotein C-III (Apoc-III), a protein that slows down the breakdown of fats called apolipoprotein C-III. By blocking the production of this protein, the medicine reduces the level of triglycerides in the blood and, as a result, fat accumulation in the body, which is expected to reduce the risk of pancreatitis.

About Waylivra

Waylivra (volanesorsen), a product of Ionis Pharmaceuticals, Inc.'s proprietary antisense technology, has received conditional marketing approval in the E.U. as a treatment for FCS. In addition, Waylivra has been granted Orphan Drug Designation by the European Medicines Agency for the treatment of FCS. Waylivra has been in-licensed by PTC from Akcea Therapeutics, Inc., a wholly-owned subsidiary of Ionis, for commercialization in Latin America.

About Familial Chylomicronemia Syndrome (FCS)

FCS is a rare disease caused by impaired function of the enzyme lipoprotein lipase (LPL) and characterized by severe hypertriglyceridemia (>880mg/dL) and a risk of unpredictable and potentially fatal acute pancreatitis. Because of limited LPL function, people with FCS cannot breakdown chylomicrons, lipoprotein particles that are 90% triglycerides. They can experience daily symptoms including abdominal pain, generalized fatigue and impaired cognitions that affect their ability to work. People with FCS also report major emotional and psychosocial effects including anxiety, social withdrawal, depression, and brain fog. The lack of treatments specifically indicated for patients with FCS means that patients must rely on a very restrictive, low-fat diet in combination with lifestyle changes and control of other secondary causes of HTG, which generally do not guarantee the necessary quality of life for those who have FCS. Diet alone does not fully control TG levels and is not sufficient to mitigate the risk of pancreatitis and enzyme replacement therapy would be ineffective in FCS due to half-life limitations.

About PTC

PTC is a science-driven, global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. PTC's ability to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need. The Company's strategy is to leverage its strong scientific expertise and global commercial infrastructure to maximize value for its patients and other stakeholders.