Business Corpus: Alnylam Stock Halted As FDA Approves First Gene-Silencing DrugAlnylam Pharmaceuticals grabbed its first Food and Drug Administration approval on Friday for a rare-disease treatment that rivals an experimental therapy from Ionis Pharmaceuticals. The post Alnylam Stock Halted As FDA Approves First Gene-Silencing Drug appeared first on Investor's Business Daily.

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Alnylam Pharmaceuticals (ALNY) grabbed a series of firsts on Friday as the Food and Drug Administration approved its first drug — a new gene-silencing treatment that targets the nerve damage component of a fatal rare disease.

Trading of Alnylam stock was halted midday on the stock market today. Ionis Pharmaceuticals (IONS), which is nearing approval for a rival drug, rose 1.4%, near 48.90. Dow Jones stock Pfizer (PFE) also is working on a treatment and saw its shares dip a fraction.

This is the first approval for a drug that treats peripheral nerve damage resulting from hereditary ATTR amyloidosis. Patisiran, now branded as Onpattro, is also Alnylam's first approved product, and is the first in a class of drugs that silence genes through a process called RNA interference.

FDA Commissioner Scott Gottlieb called the approval "part of a broader wave of advances that allow us to treat disease by actually targeting the root cause." This enables "us to arrest or reverse a condition, rather than only being able to slow its progression or treat its symptoms."

Possible Risk

Alnylam's launch could be at risk, however. A European biotech named Silence Therapeutics is seeking to block Alnylam from selling Onpattro in Portugal. The patent battle also is pending in the UK with a hearing set for early December. in addition, Silence has patents in the U.S., but hasn't asserted them.

Alnylam didn't immediately return a request for comment from Investor's Business Daily, though it said in a Securities and Exchange Commission filing that it submitted "substantive defenses" to Silence's claim in December 2017.

Patients with hereditary ATTR amyloidosis have a genetic mutation that prevents a form of protein from keeping its normal structure. This leads the protein to build up throughout the body, causing widespread difficulties, including nerve damage. That nerve damage can result in weakness, numbness or pain.

Onpattro targets the nerve damage by silencing the gene responsible for producing the disease-causing proteins. It does this through a process called RNA interference. The FDA notes RNA interference can individually turn off nearly 22,000 genes.

Rivals In Amyloidosis

Hereditary ATTR amyloidosis affects about 50,000 people worldwide. Alnylam tested Onpattro in 225 patients who received an infusion every three weeks for 18 months. Onpattro-treated patients showed improved muscle strength, sensation, reflexes and autonomic symptoms.

Ionis, too, is nearing approval for its drug known as inotersen. The FDA is set to consider Ionis' treatment on Oct. 6. Ionis and partner Akcea Therapeutics (AKCA) already have approval in Europe where they sell the drug under the brand name Tegsedi.

Pfizer is working on treating a similar condition known as transthyretin cardiomyopathy. In this condition, the buildup of abnormal protein affects the heart.

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The post Alnylam Stock Halted As FDA Approves First Gene-Silencing Drug appeared first on Investor's Business Daily.