ZUG, Switzerland & DUBAI, United Arab Emirates--(BUSINESS WIRE)-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, and taiba Middle East, a leading rare disease company based in the United Arab Emirates and covering the Middle East region, announced today that they have formed a Distribution Agreement for both ONPATTRO® and GIVLAARI®, the first-ever commercialized RNAi therapeutics, as well as another late-stage therapy in development for Primary Hyperoxaluria Type 1.

“The cooperation with taiba allows us to address the unmet medical needs of patients with rare diseases, particularly where those diseases have a higher regional prevalence. That is why it is so important that patients in the Middle East are not disadvantaged when it comes to accessing effective treatments,” said Brendan Martin, Vice President and Acting Head of Europe, Middle East & Africa, and Canada, Alnylam Pharmaceuticals. “taiba has a regional presence with operations in multiple Gulf states, and a proven track record of marketing and distributing medicines for rare diseases. Our collaboration marks an important first step in bringing our current and future therapies to those patients with urgent medical needs.”

“We are proud to collaborate with Alnylam in the Gulf states,” said Saif Al Hasani CEO of the taiba Group. “Alnylam’s portfolio of ground-breaking medicines will enhance our rare disease portfolio and enable us to offer innovative treatments to patients. Those patients with hATTR amyloidosis and other rare diseases deserve to have the earliest possible access to novel new treatments. We look forward to making this a reality, beginning with ONPATTRO and GIVLAARI.”

The Agreement between Alnylam and taiba will initially cover the Gulf states, including the Kingdom of Saudi Arabia, Kuwait, Bahrain, Qatar, Oman and the United Arab Emirates. It includes ONPATTRO, approved in the European Union (EU) in August 2018 for the treatment of hATTR amyloidosis in adults with stage 1 or stage 2 polyneuropathy; GIVLAARI, approved in the EU in March 2020 for the treatment of acute hepatic porphyria (AHP); and lumasiran, a late-stage investigational RNAi therapeutic for the treatment of Primary Hyperoxaluria Type 1 (PH1).

About ONPATTRO® (patisiran) ONPATTRO is an RNAi therapeutic that was approved in the United States and Canada for the treatment of the polyneuropathy of hATTR amyloidosis in adults. ONPATTRO is also approved in the European Union, Switzerland and Brazil for the treatment of hATTR amyloidosis in adults with Stage 1 or Stage 2 polyneuropathy, and in Japan for the treatment of hATTR amyloidosis with polyneuropathy. ONPATTRO is an intravenously administered RNAi therapeutic targeting transthyretin (TTR). It is designed to target and silence TTR messenger RNA, thereby blocking the production of TTR protein before it is made. ONPATTRO 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 polyneuropathy associated with the disease.

Important Safety Information (ISI) for ONPATTRO®

Infusion-Related Reactions Infusion-related reactions (IRRs) have been observed in patients treated with patisiran. In a controlled clinical study, 19% of patisiran-treated patients experienced IRRs, compared to 9% of placebo-treated patients. The most common symptoms of IRRs with patisiran were flushing, back pain, nausea, abdominal pain, dyspnoea, and headache. Hypotension may also occur and syncope has been reported in the post-marketing setting.

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 patisiran 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 Patisiran treatment leads to a decrease in serum vitamin A levels. Patients receiving patisiran should take oral supplementation of approximately 2500 IU vitamin A per day to reduce the potential risk of ocular toxicity due to vitamin A deficiency. Doses higher than 2500 IU vitamin A per day should not be given to try to achieve normal serum vitamin A levels during treatment with patisiran, 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. including reduced night vision or night blindness, persistent dry eyes, eye inflammation, corneal inflammation or ulceration, corneal thickening or corneal perforation).

Adverse Reactions

The most common adverse reactions that occurred in patients treated with patisiran were peripheral oedema (30%) and infusion-related reactions (19%).

For additional information about ONPATTRO, please see the full Prescribing Information.

About GIVLAARI® (givosiran) GIVLAARI is an RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) approved for the treatment of adults with acute hepatic porphyria (AHP) in the U.S. and for the treatment of AHP in adults and adolescents aged 12 years and older in the EU. In the pivotal ENVISION Phase 3 study, givosiran was shown to significantly reduce the annualized rate of composite porphyria attacks that required hospitalization, urgent healthcare visit or intravenous hemin administration at home compared to placebo. GIVLAARI is Alnylam’s first commercially available therapeutic based on its Enhanced Stabilization Chemistry ESC-GalNAc conjugate technology to increase potency and durability. GIVLAARI is administered via subcutaneous injection once monthly at a dose based on actual body weight and should be administered by a healthcare professional. GIVLAARI works by specifically reducing elevated levels of ALAS1 messenger RNA, leading to reduction of toxins associated with attacks and other disease manifestations of AHP.

Important Safety Information for GIVLAARI®

Contraindications Givosiran is contraindicated in patients with known severe hypersensitivity to givosiran. Reactions have included anaphylaxis.

Anaphylactic Reaction Anaphylaxis has occurred with givosiran treatment (