For patients with familial homozygous hypercholesterolemia (HoFH), a rare genetic disorder with incidence of 1:1,000,0001, there are now two reasons to rejoice. With the approval of Sanofi Genzyme's Kynamro (January, 2013) and Aegerion's Juxtapid (December, 2012), there are now two products which the FDA has approved for this ?ultra? orphan disease. What may not be top of mind for these patients, however, is the potentially eminent market access battle between these two products.

Juxtapid (lomitapide) is an inhibitor of the microsomal triglyceride transfer protein (MTP or MTTP) which is involved in LDL metabolism and secretion. A small-molecule, Juxtapid is manufactured as an oral pill that is indicated for daily use. Payers may cringe at the $235-295,0002 annual price tag; however the size of this patient population bodes well for its formulary coverage. The twist in the market access story comes with Kynamro, a product which more closely resembles the specialty pharmaceutical products that we?ve seen in other orphan indications.

Kynamro (mipomersen sodium) is an oligonucleotide inhibitor of apolipoprotein B (apo B), which is also involved in lipid metabolism. The language for Kynamro's indication on its label is almost identical to that of Juxtapid's label. Since its approval this past week, Kynamro has been in the spotlight, particularly for its potential price. Kynamro is administered by subcutaneous injection, which restricts its convenience from the patient's perspective. Sanofi Genzyme may be attempting to circumvent this issue by pricing Kyanmro at $176,000 per annum2.

The issue which is more likely to affect patients is whether or not both medications will be covered by payers. Orphan products have historically commanded the highest drug prices due to their small patient populations and high costs of development. But because most orphan-indicated products serve small populations and encounter fewer competitive forces as compared to other indications, payers frequently cover these agents without much pushback. This example presents an unprecedented scenario in which two orphan-indicated products are entering the market at roughly the same time. In HoFH, where global patient numbers are in the low thousands, it is not immediately clear whether it is profitable or productive for payers to support the two rival products.

While Kyanmro comes at a considerable price discount to Juxtapid, being a biologic product, it requires cold storage and may require more sophisticated manufacturing and distribution quality control. Sanofi Genzyme, who licensed Kyanmro from ISIS pharmaceuticals in 2008, has already compensated ISIS $325 million and may be obligated to compensate them up to $825 million given its regulatory approval5. Currently there are no clinical trials listed at comparing the two products head-to-head, and we do not expect any such studies in the near future; with this in mind, payer-decision making will most likely be shaped by each product's individual clinical profile. In the event that both products are covered, patient access may be driven by patient assistance programs or kickbacks from Genzyme/Aegerion. Given the growing industry-wide interest in orphan drug commercialization, companies could run the risk of encountering key market access barriers. No doubt the Kynamro and Juxtapid case will be closely watched and may shed light on future payer decision-making trends.

Jordan Pecherer is an Associate with the DRG Consulting group.

1Rader, JD et. al., Monogenic hypercholesterolemia: new insights in pathogenesis and treatment. J Clin Invest. 2003 June 15; 111(12): 1795?1803.
2Rockoff, J.D. (Jan. 2013) The Big Business of Orphan Drug. Wall Street Journal, Accessed online at
3Kyamro Package Insert. Accessed 2/7/2013 from Drugs@FDA
4Juxtapid Package Insert. Accessed 2/7/2013 from Drugs@FDA
5Press Release: ?Genzyme, Isis announce results of two mipomersen phase 3 studies?

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