The 5 Most Expensive Drugs In US: What You Should Know

The upward trajectory of prescription drug prices in the U.S. has drawn the ire of lawmakers. Prices of drugs are staggeringly high in the U.S. when compared to other developed countries such as Japan and in Europe.

House lawmakers are discussing a plan that could work to curb high drug prices.

Some of the proposals include allowing the Secretary of Health and Human Services to negotiate with pharma companies on the prices of drugs covered by Medicare.

If no agreement were reached, the decision would rest with the Government Accountability Office, which is given the prerogative to fix prices based on the median price of a drug in other countries.

Pharma executives are pushing back, reasoning that the prevailing prices are justified given the billions of dollars invested in R&D.

Here's a look at five of the most expensive drugs in the U.S.

1. Zolgensma

Novartis AG NVS's gene therapy Zolgensma is a gene therapy for pediatric patients with spinal muscular atrophy, or SMA — a neurodegenerative disease that affects nerves controlling the muscles and ultimately causes loss of muscle function.

Zolgensma, when delivered as a one-time infusion, functions by replacing the missing or defective SMN1 gene with a functional copy that is capable of making SMN protein that improves motor-neuron function and survival.

It was approved by the FDA May 24. Zolgensma is priced at $2.125 million, but Novartis has facilitated payment by some insurers in annual installments of $425,000 over five years.

Notwithstanding the astronomical price tag, some still consider it as a cost-effective option, given that it is a one-time treatment.

2. Luxturna

Spark Therapeutics Inc ONCE, which has agreed to be acquired by Roche Holdings AG Basel ADR RHHBY, is credited with the first-ever gene therapy approved in the U.S.

Luxturna, an AAV vector-based one-time gene therapy, is indicated for the treatment of confirmed biallelic RPE65 mutation-associated retinal dystrophy, an inherited retinal disorder that leads to vision loss. It was approved in December 2017 and launched commercially in 2018.

The gene therapy is priced at $425,000 per eye for a total cost of $850,000 per patient.

See also: ContraVir Rebounds From Record Low On Positive FDA Feedback

3. Myalept

Myalept was approved by the FDA in February 2014 as an adjunct to diet as a replacement therapy for the treatment of complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy.

Leptin is a hormone made by adipose cells and the small intestine that helps regulate energy balance by inhibiting hunger. Deficiency of this hormone causes severe obesity.

Myalept is marketed by Aegerion Pharmaceuticals, a subsidiary of Novelion Therapeutics Inc NVLN. It is administered as an injection once-daily at the same time everyday.

Assuming patients typically use 14 vials per month and each vial is priced at $4,633, the drug costs $778,344 per year, according to Good Rx.

4. Folotyn

Folotyn, which is now under the stable of Aurobindo Pharma USA Inc's subsidiary Acrotech Biopharma, is indicated for the treatment of patients with relapsed or refractory peripheral T-cell lymphoma.

It was approved by the FDA in September 2009.

Folotyn was originally developed by Allos Therapeutics, which was acquired by Spectrum Pharmaceuticals, Inc. SPPI in 2012. Recently, Spectrum sold the drug to Acrotech.

The drug costs $745,785 annually.

5. Soliris

Alexion Pharmaceuticals, Inc. ALXN's Soliris has been approved for three indications. It was approved for paroxysmal nocturnal hemoglobinuria in March 2007 and for atypical hemolytic uremic syndrome in September 2011. In October 2017, it was approved for use in patients with generalized myasthenia gravis, who are anti-acetylcholine receptor antibody-positive.

Soliris carries an expensive price tag of $678,392, according to Good Rx.

The list has been compiled based on data from GoodRx,  a website and mobile app that tracks prescription drug prices in the U.S. Good Rx ranks drugs by their annual costs for a typical course of treatment.

Related Link: 6 Gene Therapy M&A Targets On The Radar