VentureBeat

(UPDATED: See below.)

Almost two weeks ago, Genentech angered doctors and elderly patients when it announced plans to restrict access to Avastin, a cancer drug that doubles as an unapproved, but quite inexpensive, treatment for eye disease. In those conditions, which can lead to encroaching blindness when left untreated, Avastin competes with a much more costly Genentech drug called Lucentis. (For background and our take on the situation, click here.)

Now the giant biotech appears to be taking a few steps back from the precipice. In an open letter posted on its site yesterday, Genentech said it will postpone plans to bar Avastin sales by “compounding pharmacies” — which can safely divide up a large vial of the drug into the tiny doses needed for intraocular injection (see photo at left) — by a month, giving retinal specialists and patients until Jan. 1 to find other suppliers. Perhaps more important, Genentech said it would continue to make Avastin available to compounding pharmacies should the FDA grant it “legal and regulatory authority to do so.”

Genentech also offered a more detailed explanation for its original decision to limit Avastin distribution, noting that the FDA had warned a compounding pharmacy about the sterility and repackaging of the drug. Worse, at least from the big biotech’s perspective, an FDA inspection of a Genentech manufacturing facility raised questions about the suitability of some Avastin production lots for use in the eye due to a “higher visual inspection standard.” (More on that when I know more.) Genentech says that to “resolve” the FDA’s concerns, it destroyed four batches of Avastin, amounting to 350,000 vials with a market value of more than $200 million — a vastly inflated estimate of the company’s actual loss, given that the marginal production cost to Genentech is almost certainly far lower than that.

To be fair, the issues here are complex, although it’s also important to bear in mind that we’re only hearing one side of the story — the FDA doesn’t comment on regulatory matters. If we take Genentech’s version of events at face value, then the company does appear to face additional regulatory scrutiny related to the ocular use of Avastin, with the potential for further unexpected losses if inspectors require the destruction of additional production lots. It’s hard not to sympathize with management under such conditions.

But there’s still something missing in this explanation. Even with the compounding pharmacies out of the picture, no one expects Avastin use in eye disease to go away, since ophthalmologists will still be able to get it from hospitals or other doctors. As a result, Genentech’s regulatory risk doesn’t really go away, either — if the FDA is concerned about Avastin’s suitability for ocular use when, say, half of elderly patients with wet age-related macular degeneration are using the drug, is the agency really going to be that much less concerned if only one-quarter of these patients are using Avastin? That’s still a lot of people.

So while Genentech’s concern about potential problems with the FDA certainly can’t be dismissed, it’s still not really sufficient to explain their actions. The only way for the company to really placate a safety-obsessed FDA — Genentech’s implicit characterization, not mine — would be either to declare total war on ocular Avastin use or to improve Avastin manufacturing so that the product would satisfy the FDA’s alleged concerns. So far, of course, Genentech hasn’t done either, although its allusion to getting FDA permission to supply compounding pharmacies suggests that it may be weighing the latter. If so, that’s all to the good.

Still, the company’s first instinct was clearly to inconvenience patients, many of them desperate, with a move that might also improve its bottom line, instead of taking steps that would clear up the problem for good, albeit at some expense and potential harm to future Lucentis sales. That still says a great deal about the company’s claimed commitment to “the best interests of patients.”

Hat tip to the WSJ health blog

UPDATE: David Williams raises some similar questions at the Health Business Blog, and apparently manages to dig up the FDA warning letter to the compounding pharmacy as well.

(UPDATED: See below.)

Committing your biotechnology giant to “the best interests of patients [and] the medical profession,” as Genentech CEO Arthur Levinson does on its his company’s Web page, is certainly a fine sentiment. When you subsequently decide to restrict use of a drug used by many elderly individuals to ward off encroaching blindness, however, you probably shouldn’t be surprised if people begin wondering whether that commitment is anything more than an empty slogan.

Restricting access to such a drug, of course, exactly what Genentech did last week, when it announced new limitations on the distribution of its cancer drug Avastin. That drug, a certifiable hit in treating colon, lung and breast cancer, has recently taken on a new role as an apparently effective — and dirt cheap — way of treating wet age-related macular degeneration, a progressive eye disease of the elderly that generally leads to near-total blindness.

The problem for Genentech is that it also sells a newer and far more expensive AMD drug called Lucentis, which runs close to $2,000 per monthly shot. By contrast, Avastin — a close biochemical cousin to Lucentis — is priced for use in far larger doses as a cancer treatment, so the tiny amount needed for injections into the eye costs only about $40 a shot.

For more than two years, retinal specialists have obtained Avastin through compounding pharmacies, which can safely divide up a large vial of Avastin into syringes for individual eye injections. Last Thursday, however, Genentech announced that it would no longer permit compounding pharmacies to obtain the drug. (Avastin is still available through wholesale distributors.) The move will almost certainly crimp the availability of the drug for the roughly half of elderly AMD patients who have been using it as an alternative to Lucentis. That drug, even when covered by Medicare or private insurance, can still cost patients $400 or more in co-payments for every shot.

Genentech dressed its decision in the corporate doublespeak that’s long been associated more with Big Pharma than Big Biotech, saying it was prompted by FDA concerns about the sterility and repackaging of Avastin. (Last time I checked, there were no actual case reports of sterility problems with Avastin use in AMD — and if there are any now, surely Genentech would have cited them.) The company also notes in its letter that “Avastin has not undergone any formal, randomized, controlled clinical trials for ocular use.” This is true, but it’s pretty rich to hear that objection coming from Genentech, which has refused to cooperate with a head-to-head trial of Avastin and Lucentis planned by the National Eye Institute.

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