Rocco Verducci

What is GSK Thinking With Its Latest NDA?

Eben,

As an FYI - Promacta was approved by the FDA today.

Ciao,

Novartis Buys Protez For Super Anti-Biotic Defense

Eben,

The carbapenem class of antibiotics has demonstrated excellent efficacy against a wide range of bacteria and make it an very good choice for presumptive therapy for infection.

Novartis has actually limited their financial exposure by structuring a deal that only has them paying $100M up front with the rest to be paid only if the antibiotic meets certain developmental milestones. Additionally, Novartis's scientists have no doubt done their due diligence with respect to the initial payment and the expectations are probably very high for this antibiotic.

Ciao

Indevus Pharmaceuticals: Nebido Has Lost Its Libido

Steve,

The FDA, by design, exercises extreme caution with respect to their evaluation and approval of drugs since the health of every American is potentially at risk. Additionally, their judgment is devoid of the commercial zeal that investors have about a particular product. If we had to shoulder the responsibility of evaluating a drug and observing the consequences of prematurely approving a drug that should have had a more rigorous screening, I truly believe that most would err on the side of caution.

There are presently testosterone products that are available that are delivered through safer delivery systems (i.e. transdermally). What I would be more concerned with is the potential that androgen therapy has for increased risk of benign enlargement of the prostate or that it may promote the growth of existing cancer of the prostate. However, the once every three month injectable regimen does confer a patient-centric advantage in terms of convenience.

Although the indication for Nebido is for hypogonadism (Common effects of hypogonadism include decreased sexual desire, erectile dysfunction, muscle loss and weakness, depression, as well as an increased risk of osteoporosis), the name Nebido suggests that its primary function will be to elevate the libido in men. I do believe that the phosphodiesterases (e.g. Viagra, Levitra and Cialis) will still maintain their market preeminence and support by physicians since they have specificity for the primary problem associated with erectile dysfunction (ED) without the potential of increasing the risk associated with androgens.

If the parent company continues on its track of developing innovative compounds, it is likely that a large pharma or biotech company will either partner with them or enter into some equity agreement.

Ciao,

MS Drug Fampridine Sends Acorda Stock Way Up

Eben,

The mechanism of action for this compound is unique. Since it doesn't approach MS from an auto-immune vantage point it would make this a natural as an adjunctive therapy.

The unknown is who the Acorda marketing partner would be. The company has a 14-person internal sales force and a 160-member contract sales force selling Zanaflex and this doesn't appear to be the critical mass necessary to provide the educational and marketing effort to have Fampridine-SR recognized as a primary treatment for MS.

At the end of the day, you need to have an innovative molecule coupled with a great company to extol the benefits (and risks) of a particular compound and to make it commercially viable.

Ciao,

What is GSK Thinking With Its Latest NDA?

Eben,

Please accept my apologies if you inferred that I was chiding you. That was not my intent. I supply the additional information as an FYI.

ITP can be divided into two different forms

• Acute ITP starts suddenly and usually follows a viral illness in a child. Acute ITP may require no treatment, especially if the platelet count does not fall too low and there is little bleeding. It usually improves spontaneously and, in children at least, rarely comes back.*

• Chronic ITP develops over time, is long lasting and more common in adults. It may not need treatment if the platelet level doesn't pose a significant risk of -bleeding. *

Both are treated similarly. However in the chronic setting if the platelets fall dramatically, then a product with the promise of a Promacta may help restore platelets to a medically acceptable level. There are a wide range of medical conditions where thrombocytopenia may be a critical concern (e.g. intracranial hemorrhage) or it may be a medical state that precludes the use of life-saving drugs (e.g. chemotherapy, interferon therapy or antiviral therapy) for disease entities such as cancer, HIV and Hepatitis. Theoretically, if a pharmaceutical were to have the properties of remarkably stimulating platelet synthesis, then it may allow the critical use of life-saving drugs in a patient population whose low platelet count (in the past) has been an exclusion criterion.
The vying for a “chronic” indication for ITP would not be plausible from an indication perspective since it would necessitate the need to have long-term studies completed at the time that the NDA was filed. Additionally, this would delay any potential use of a product in a patient population that has a bona fide need for its therapeutic benefit today. What may be more achievable is to have application for the “episodic” or “acute” aspect of a chronic condition. This may very well be the rationale for the drug that you have inquired about.

As to your question as to the rationale for an “acute” medication treating a “chronic” condition; this paradigm of use is no different from how asthma is medically managed. People with asthma use pharmaceuticals to control inflammation and vasoconstriction which are part of their chronic state. However, when they have an acute attack, they augment their management of asthma with a rescue inhaler (albuterol). As valuable as their rescue inhalers are, they are intended to be used only when there is an acute attack of their chronic condition.

In any event, if you add 98 cents to the aforementioned, you should have one dollar.

Ciao,

• footnote - www.netdoctor.co.uk/di...
• P.S. I enjoy reading your column.

What is GSK Thinking With Its Latest NDA?

Eben,

It might be prudent to examine the total body of clinical work before rendering a negative opinion on Promacta or GSK. The FDA's outside advisory board of learned clinicians opined a positive outlook about Promacta with respect to ITP. Historically, the FDA follows the guidance of the advisory board.

Sincerely,