Therapy Areas: Inflammatory Diseases
Alnylam Pharmaceuticals and Taiba Group Partner to Commercialize RNAi Therapeutics in the Gulf States
8 July 2020 - - Swiss RNAi therapeutics company Alnylam Pharmaceuticals, Inc. (NASDAQ: ALNY) and UAE-based rare disease company taiba Middle East 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 companies said.

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 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 ; and lumasiran, a late-stage investigational RNAi therapeutic for the treatment of Primary Hyperoxaluria Type 1.

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. 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 for ONPATTRO

Infusion-related reactions 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.

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).

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

GIVLAARI is an RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) approved for the treatment of adults with acute hepatic porphyria in the US 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.

Anaphylaxis has occurred with givosiran treatment (
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