GENZYME’s CEREDASE ANNUALIZING AT $80 MIL. -- FIRM’s 1991 SALES $20 MIL. HIGHER THAN EXPECTED; MOST GAUCHER’s PATIENTS PAYING UNDER $2,000 ANNUALLY, FIRM SAYS

Genzyme's Ceredase (alglucerase) enzyme for treatment of Type I Gaucher disease is selling at an annualized rate of approximately $80 mil., the company said in a Feb. 26 statement of 1991 financial results. The drug was introduced last April. Sales appear to be considerably higher than anticipated by the firm. Genzyme previously had estimated that 1991 sales for Ceredase would be about $38 mil. ("The Pink Sheet" Jan. 27, p. 10). At annualized rate of $80 mil., Cederase sales are running at about $6.7 mil. a month, or approximately $60 mil. for the product's first nine months on the market. And in fact, Genzyme says it exceeded its revenue goal for the year by "more than $20 mil.," almost all of which could have been derived from Cederase. The company reported total sales for the year of $121.7 mil., more than double 1990 results. While Cederase revenues are running at a healthy pace, the company continues to defend pricing. Most Ceredase patients are paying less than $2,000 a year for the treatment, the company says. Speaking at a Feb. 27 session of a National Health Lawyers Association meeting on reimbursement issues in Washington, D.C., Genzyme Director of Reimbursement for Biotherapeutics William Aliski noted that actual out-of-pocket expenses are topping out at $2,000 or less for the majority of Cederase patients, as "67% of our patients have a stop-loss of $2,000 or less. That is to say very few patients, [about] a third of the patients, are paying in excess of $2,000 on an annual basis for Ceredase." Overall, "80% of our [Ceredase] patients have a copay of 20% or less," Aliski added. The company told a Jan. 21 hearing of the Senate Judiciary/Antitrust Subcommittee on orphan drugs that the first year of intensive therapy with Ceredase for severely ill patients costs about $250,000 to $300,000 but then decreases as doses taper down. Aliski also said that Genzyme has established a program whereby it will "provide free drug to patients who otherwise don't have insurance resources...Basically, if you don't have insurance, you can't afford the dosages that most people get... We have this program for people to apply to it if they have no other resources and no insurance and they're not eligible for public health programs, we provide this drug free of charge." Asked to estimate the number of patients who receive Ceredase free, Aliski replied: "Less than 5%." Genzyme moved firmly into the black for the year, with net of $19.6 mil. reversing last year's loss of $27.1 mil. Company Chairman Henri Termeer noted that Genzyme will have eight products in clinical trials this year, "which positions us for continued growth."