BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) is ready to break above the $1 billion revenue barrier in 2016. This is a feat that marks a major hurdle for many biotech and emerging pharmaceutical companies. Now the company’s stock has gained on hopeful data on its hemophilia treatments. 24/7 Wall St. might caution that this data is very preliminary and that it will be quite some time before it is clear whether BioMarin’s investigational gene therapy treatment for hemophilia A will be a home run.

What really matters here is that hemophilia is one of the conditions that are the most expensive to treat. The average medical costs for hemophilia can be massive for insurers and for people who suffer from it. Hemophilia-Information.com notes that hemophilia therapy is among the most expensive in the world, with a total annual per-patient cost ranging from $60,000 to as much as $1,000,000. The site says:

Many patients are on a prophylactic treatment plan requiring an intravenous(IV) infusion 3 times per week, for life. One infusion for an adult weighing 150 lbs. is approximately $3000 for the medication alone. The average cost for adult patients on a prophylactic regiments at this rate is currently $468,000 annually.

BioMarin said that its BMN 270 showed positive Phase 1/2 clinical data as an investigational gene therapy treatment for hemophilia A. BMN 270 has already received an orphan drug designation from the U.S. Food and Drug Administration (FDA) and from the European Commission. The company noted that Phase 3 trial design preparation and high volume manufacturing plans are underway.

Patients with hemophilia A are unable to produce enough functional Factor VIII to prevent bleeding. These patients are currently treated with prophylactic or on-demand infusions of plasma-derived or recombinant Factor VIII. How BioMarin is differentiating its BMN 270 is by saying that the treatment was designed to address the underlying genetic defect that prevents the expression of functional Factor VIII. This is done by using an adeno-associated virus vector to deliver a functional copy of the factor VIII gene to the cells of each patient with the aim of a single infusion of BMN 270 providing a long-lasting increase in Factor VIII levels.

BioMarin’s revenues have grown and grown. They are also expected to grow this year and next, to $1.09 billion and to $1.34 billion, respectively. Interestingly enough, BioMarin is still expected to post operating losses. Thomson First Call has its consensus estimate at −$2.48 EPS for 2016 and −$1.31 EPS for 2017.

Hank Fuchs, M.D., chief medical officer at BioMarin, said:

We are encouraged by this early data on BMN 270 and the trend we are seeing in increasing Factor VIII levels over time.  BMN 270 could have the potential to reduce and possibly eliminate the need for infusions of Factor VIII.

BioMarin also included a quote from John Pasi, Ph.D. F.R.C.P, who is Professor of Haemostasis and Thrombosis at Barts and the London School of Medicine and Dentistry. Pasi is listed as the primary investigator for the BMN 270 Phase 1/2 clinical trial. He said:

If BMN 270 allows hemophilia A patients to maintain around 5% of normal levels of Factor VIII, it could have a real and meaningful clinical benefit by reducing the need for Factor VIII infusions and spontaneous bleeds. I am looking forward to further assessing the data over the 16 weeks and beyond in this ongoing study.

It is too soon to know anything about potential pricing for BMN 270. After all, it is still in Phase 1/2 trials and later trial data (and costs) remains to be seen. There are also no assurances that the treatment data will be uniform for larger trial numbers, nor any assurances that it will be approved in the United States or Europe.

It is no secret that health care costs have been rising. When insurance companies get just one hemophilia case on their plans, it can literally take the profits of hundreds (or more) insured cases to offset the high expenses of treating it. Having costs be in the hundreds of thousands per year, let alone being as much as $1 million per year, for just one patient is a significant cost in the grand scheme of things. That also means that it can represent a huge opportunity for the companies making the next steps in treating such expensive conditions.

BioMarin shares were last seen up 5.5% to $91.15 on Wednesday, and their 52-week trading range is $62.12 to $151.75. The company’s market cap is $14.7 billion, and the consensus analyst price target is just over $115.

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