In an eagerly awaited milestone for the company – and for Belgian biotech – the FDA recently approved ThromboGenics NV's biologic drug Jetrea (ocriplasmin) for treating symptomatic vitreomacular adhesion (VMA).

Following a 10-0 vote by the FDA's Dermatologic and Ophthalmic Drug Advisory Committee in favor of its approval earlier this year, there was little doubt that Jetrea would get over the line. (See BioWorld Today, Jul. 27, 2012.)

Crucially, however, there was no sting in the tail, in terms of the label. "It's a broad label. It is a label with no restrictions," Patrik De Haes, CEO of Leuven-based ThromboGenics, told BioWorld International.

The FDA decision followed a priority review, conducted at the instigation of the agency. ThromboGenics initially had sought a standard review, and submitted a biologics license application on Dec. 23, 2011. Following feedback from the FDA, it withdrew the file and resubmitted on April 17.

The company will commercialize the product in the U.S. itself and is preparing to launch it in January. It is in the process of assembling a sales force comprising 28 field representatives and 16 reimbursement executives. "We have identified most of the people," De Haes said. "We plan to forward them contracts by the end of the week."

ThromboGenics previously guided a U.S. price of $3,000 per vial – the treatment is administered as a single, intravitreal injection – although, based on market feedback, "the price point could be higher," De Haes said. In Europe, likewise, the company expects to exceed its previously guided price of more than €2,000 (US$2,620) per vial.

Jetrea is still under review at the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) – the file was accepted exactly one year ago. "The CHMP decision should come by late January," De Haes said. "The drug could be on the [European] market by late next April," he added. That would trigger a €90 million milestone payment from the Alcon unit of Basel, Switzerland-based Novartis AG, which licensed ex-U.S. rights earlier this year, in a deal worth up to €375 million plus royalties. (See BioWorld Today, March 19, 2012.)

ThromboGenics is not issuing firm guidance on sales until it has a few quarters under its belt, but it expects the product to attain "several hundred millions" in sales for VMA. Writing in a research note, Jefferies analyst Peter Welford was even more bullish, forecasting peak worldwide sales of $875 million, based on a penetration of 40 percent of the patient population.

The target audience for the product is tightly defined, comprising 2,000 specialists in the U.S. "Over 90 percent know about the product," De Haes said, an estimate based on market research the company commissioned.

The publication of Phase III data in the Aug. 16, 2012, issue of The New England Journal of Medicine (in a paper titled "Enzymatic vitreolysis with ocriplasmin for vitreomacular traction and macular holes") will enhance the scientific credibility of the product, De Haes said. "We offer an alternative to surgeons for severe patients and offer the only opportunity available for patients who are less severe."

VMA is caused by contraction of the eye's vitreous humor or gel that occupies the cavity between the lens and the retina. The presence of residual adhesions can result in tears in the macula of the retina, leading to visual distortion and even blindness. Jetrea, which comprises a truncated form of a serine protease enzyme called plasmin, dissolves the protein matrix responsible for the adhesions.

ThromboGenics' founder and chairman, Desire Collen, a veteran biomedical scientist who previously co-discovered tissue plasminogen activator (tPA), had initially explored the potential of microplasmin in cardiovascular indications. The concept of applying the molecule to ophthalmic indications was originally pioneered by U.S. retinal surgeons.

ThromboGenics and Alcon now are working on additional indications for the drug, including diabetic retinopathy and age-related macular degeneration (AMD). Data from a Phase II study in AMD are due in the second quarter of next year.

Shares in ThromboGenics (BRUSSELS:THR) gained €1.40, or almost 4 percent, last Thursday to close at €38.40, the modest gain a reflection of the fact that investors had long priced in the approval. The stock has doubled since the start of the year. The company is now valued at €1.375 billion, which places it at the front rank of European biotechnology firms.

ThromboGenics reported €186.1 million in cash at June 30 and, because of a €75 million up-front payment from the Alcon deal, will report a profit this year. The likely €90 million milestone linked to a European approval next year will keep the company in the black, and thereafter, product sales should ensure that it will become sustainably profitable.

As well as investing in its own pipeline – which, barring a preclinical oncology program, is now exclusively focused on ophthalmology indications – ThromboGenics will be on the lookout for other possibilities in ophthalmology. "We definitely will keep some of the cash earmarked to invest in those kind of assets," said Chris Buyse, chief financial officer.