Alnylam Continues To Deliver On RNAi Pipeline With Alternative Treatment Option For Rare Disease


  • Alnylam had obtained FDA approval for its drug GIVLAARI to treat patients with acute hepatic porphyria.
  • The FDA approved GAVLAARI a few months early based on the drug's ability to reduce porphyria attacks in AHP patients by 70% compared to placebo.
  • ONPATTRO is the company's first RNAi drug to be approved by the FDA which treats polyneuropathy in patients with hATTR amyloidosis.
  • Alnylam generated $46.1 million in sales of ONPATTRO in Q3 of 2019, ongoing phase 3 APOLLO-B to be used to expand market opportunity going after hereditary and wild-type ATTR cardiomyopathy patients.
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Alnylam Pharmaceuticals (NASDAQ:ALNY) had obtained FDA approval for its drug GIVLAARI to treat patients with acute hepatic porphyria (AHP). This is a very rare disease, which means it can be priced at higher price. The company has a solid pipeline being advanced to treat a host of rare diseases using its RNAi technology. Not only that, but GIVLAARI was the second drug to be approved by the FDA for Alnylam within the last 16 months. Alnylam continues to make progress with its RNAi technology platform. That's why I believe it is a good buy.

FDA Approval Of GIVLAARI Is Not Blockbuster Status, But Will Still Produce Solid Revenue

Alnylam received FDA approval of GIVLAARI to treat patients with acute hepatic porphyria. Acute hepatic porphyria is an ultra-rare disease in which patients lack a particular enzyme in the liver causing a host of devastating symptoms. The deficiency of the enzyme can happen in 1 of 4 types of AHP. They are:

  • Acute intermittent porphyria (AIP)
  • Variegate porphyria (VP)
  • Hereditary coproporphyria (HCP)
  • ALA dehydratase-deficiency porphyria (ADP)

This is a terrible disease, because the missing enzyme for one of the four particular AHP types causes a host of symptoms. They can range anywhere from unexplained abdominal pain, nausea, vomiting, diarrhea and a multitude of many other symptoms you can think of. The FDA approved the drug as quickly as it possibly could. To this, I'm referring to the fact that the drug was approved by the agency after only 4 months of the NDA being submitted. The Priority Review would at least get the drug looked at 6 months after an NDA submission. The FDA approving the drug a few months in advance is good news for these patients and for Alnylam itself. For the patients, it primarily boils down to the nasty porphyria attacks they experience constantly. This has a major impact on their quality of life. The big improvement for patients treated with GIVLAARI, in the late-stage study, is the biggest reason why the drug was approved much quicker than anticipated. Positive results came from the phase 3 ENVISION study. This was where a total of 94 patients with AHP were recruited into the double-blind randomized, placebo controlled study. It was noted that patients treated with Alnylam's GIVLAARI had achieved on average 70% fewer porphyria attacks compared to placebo. As I alluded to above, these porphyria attacks are devastating for the patient. The ability to see such a massive reduction in attacks should be highly welcomed. During the study, it was noted that there were two highly common adverse reactions. This involved about 20% of the patients in the study with either injection site reactions or nausea. While there are some side effects taking the drug, the ability for patients to see a reduction of attacks in that capacity should be highly welcomed.

Additional FDA-Approved Product Reduces Risk

GIVLAARI is the second drug to be approved by the FDA for Alnylam. The company had already received FDA approval for another RNAi product in the pipeline now marketed as ONPATTRO. ONPATTRO was approved to treat polyneuropathy in patients with hereditary transthyretin-mediated (hATTR) amyloidosis. The thing is that hATTR is a rare genetic disease where misfolded proteins cause amyloid deposits. These amyloid deposits damage any organ in the body. In the case of this drug, it was approved to deal with the damage to the nerves of the body causing polyneuropathy. Polyneuropathy meaning damage occurring to the peripheral nerves. There are a few problems that patients experience with polyneuropathy which can include weakness of the body, numbness and a type of burning pain. It is a rare disease. Therefore, it only affects approximately 50,000 patients worldwide. ONPATTRO seems to be selling well, as Alnylam generated Q3 2019 global sales of $46.1 million for it. The thing is that Pfizer (PFE) also has its own hATTR drug approved known as Tafamidis. Tafamidis goes after the cardiomyopathy patients with hATTR (this deals with damage of the cardiovascular system). This group is a bit more prevalent compared to the polyneuropathy type. Pfizer is a competitor, but even then, Alnylam is not sitting still. That's because it had initiated a phase 3 study known as APOLLO-B. The reason for doing so is to go after hereditary and wild-type ATTR cardiomyopathy patients. The results of this study will be important in determining if Alnylam can ultimately update the label to include these other types of hATTR populations. Pushing this aside as data is not expected for a while, I expect ONPATTRO sales to continue to climb. Sales will grow because ONPATTRO had some initial sales in Japan in the past quarter. In addition, the company had filed an NDA for potential approval to market its drug in Brazil. Additional achievements with ONPATTRO include a commercial launch in Canada and approval obtained for marketing the drug in Switzerland. Alnylam is in very good shape, as it has $1.74 billion in cash and investments to fund its pipeline and launch its drugs in new territories.


Alnylam having received FDA approval of GIVLAARI to treat patients with acute hepatic porphyria is good news particularly for patients. Consider that these patients experience a multitude of symptoms depending upon 1 of the 4 subtypes of AHP. Symptoms can range from nausea, vomiting, limb weakness and pain. In more severe cases, it can even cause life-threatening issues. One example of a life-threatening problem with AHP involves something known as Tachycardia, where your heart beats more than 100 times per minute. As you can imagine, these patients have a diminished quality of life. But wait a second, is there any other treatment out there for AHP? Treatment can be done with carbohydrate loading, IV glucose, or IV hemin. These can be given when a patient experiences a porphyria attack or in between attacks. Before GIVLAARI, there were no treatments approved to prevent debilitating attacks and treat chronic symptoms of AHP. The risk with this drug comes in terms of how well it does on the market. That's because it is expected to cost about $442,000 per year with discounts. I don't think that will be a problem, though. That's because these patients really need a new treatment option. Especially, when you consider that GIVLAARI is used to treat the underlying issues associated with attacks of AHP. This was evidenced in the sharp reduction of porphyria attacks by 70% compared to placebo. I believe Alnylam is in good shape. Not only does it have the most recent FDA approval with GIVLAARI to treat AHP, but it continues to expand its label for its first RNAi approved treatment ONPATTRO in patients with polyneuropathy with hATTR.

This article is published by Terry Chrisomalis, who runs the Biotech Analysis Central pharmaceutical service on Seeking Alpha Marketplace. If you like what you read here and would like to subscribe to, I'm currently offering a two-week free trial period for subscribers to take advantage of. My service offers a deep-dive analysis of many pharmaceutical companies. The Biotech Analysis Central SA marketplace is $49 per month, but for those who sign up for the yearly plan will be able to take advantage of a 33.50% discount price of $399 per year.

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