Alnylam Announces Unaudited Fourth Quarter 2018 Global Revenues for ONPATTRO® (patisiran) and Provides Additional Commercial Updates
− Achieved Fourth Quarter 2018 ONPATTRO Unaudited Global Net Product
− Over 200 Patients on Commercial ONPATTRO in U.S. and EU from Launch Through Year-End 2018 –
− Maintained Strong Balance Sheet with Unaudited Year-End Cash and
Investments Balance of Approximately
ONPATTRO global net product revenues (unaudited) for the fourth
quarter of 2018 were
- As of year-end 2018, over 200 patients in the U.S. and EU were receiving commercial ONPATTRO treatment, and approximately 550 total patients worldwide, including patients on commercial drug and patients in clinical studies and in the Company’s global Expanded Access Program (EAP), were being treated with patisiran.
In the U.S., a total of 250 Start Forms were submitted as of year-end
2018. Of these, approximately 50 percent were from patients previously
treated on the ONPATTRO EAP.
- The Start Forms came from a diverse range of prescribing physician specialties, including 44 percent neurologists, 35 percent cardiologists, and 21 percent from other specialties.
For Start Forms received, 62 percent of patients were covered by
Medicare, 32 percent were covered by commercial insurers, and 6 percent were covered by other government insurers.
Significant progress has been made with value-based agreements (VBAs)
in the U.S. and with market access efforts in the EU. Since launch,
Alnylam has completed full VBAs with
Harvard Pilgrim Healthcare, Humana, and another top five U.S. payer. Additional VBAs are under negotiation with over 15 other commercial payers with the potential to cover over 90 percent of commercial lives in the U.S.
“2018 was a landmark year for Alnylam, marked by the approval and launch
in the U.S. and EU of ONPATTRO, heralding the arrival of RNAi
therapeutics as a whole new class of medicines. Our unaudited fourth
quarter 2018 global net product revenues of
In addition, the Company today reported that at
About ONPATTRO® (patisiran)
Patisiran, based on Nobel Prize-winning science, is an intravenously administered RNAi therapeutic targeting transthyretin (TTR) for the treatment of hereditary ATTR amyloidosis. It is designed to target and silence TTR messenger RNA, thereby blocking the production of TTR protein before it is made. Patisiran blocks the production of TTR in the liver, reducing its accumulation in the body’s tissues in order to halt or slow down the progression of the disease. In
Important Safety Information
Infusion-related reactions (IRRs) have been observed in patients treated with ONPATTRO. In a controlled clinical study, 19 percent of ONPATTRO-treated patients experienced IRRs, compared to 9 percent of placebo-treated patients. The most common symptoms of IRRs with ONPATTRO were flushing, back pain, nausea, abdominal pain, dyspnea, and headache.
To reduce the risk of IRRs, patients should receive premedication with a corticosteroid, paracetamol, and antihistamines (H1 and H2 blockers) at least 60 minutes prior to ONPATTRO infusion. Monitor patients during the infusion for signs and symptoms of IRRs. If an IRR occurs, consider slowing or interrupting the infusion and instituting medical management as clinically indicated. If the infusion is interrupted, consider resuming at a slower infusion rate only if symptoms have resolved. In the case of a serious or life-threatening IRR, the infusion should be discontinued and not resumed.
Reduced Serum Vitamin A Levels and Recommended Supplementation
ONPATTRO treatment leads to a decrease in serum vitamin A levels. Supplementation at the recommended daily allowance (RDA) of vitamin A is advised for patients taking ONPATTRO. Higher doses than the RDA should not be given to try to achieve normal serum vitamin A levels during treatment with ONPATTRO, as serum levels do not reflect the total vitamin A in the body.
Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g. night blindness).
The most common adverse reactions that occurred in patients treated with ONPATTRO were respiratory-tract infection (29 percent) and infusion-related reactions (19 percent).
About LNP Technology
Alnylam has licenses to Arbutus Biopharma LNP intellectual property for use in RNAi therapeutic products using LNP technology.
RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines, known as RNAi therapeutics, is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam's RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing proteins, thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.
Alnylam (Nasdaq:ALNY) is leading the translation of RNA interference (RNAi) into a new class of innovative medicines with the potential to improve the lives of people afflicted with rare genetic, cardio-metabolic, hepatic infectious, and central nervous system (CNS) diseases. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach for the treatment of a wide range of severe and debilitating diseases. Founded in 2002, Alnylam is delivering on a bold vision to turn scientific possibility into reality, with a robust discovery platform. ONPATTRO® (patisiran) lipid complex injection, available in the U.S. for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults, is Alnylam’s first U.S.
Alnylam Forward Looking Statements
Various statements in this release concerning Alnylam's future expectations, plans and prospects, including, without limitation, Alnylam's views with respect to the potential for RNAi therapeutics, its unaudited global net ONPATTRO revenue for the fourth quarter of 2018, its unaudited cash, cash equivalents and marketable debt securities, and restricted investments balance, excluding equity securities, as of
With the exception of ONPATTRO (patisiran), none of Alnylam’s
investigational therapeutics have been approved by the
Alnylam Pharmaceuticals, Inc.
Christine Regan Lindenboom
(Investors and Media)