[Ip-health] 11th Circuit ruling in an Orphan Drug Act case shows how screwed up the Act is

James Love james.love at keionline.org
Thu Oct 7 08:47:11 PDT 2021

This is the ruling in
XAVIER BECERRA, Secretary of Health and Human Services, U.S. DEPARTMENT OF
and Drug Administration, U.S. FOOD AND DRUG ADMINISTRATION,
Defendants – Appellees,

Intervenor-Defendant – Appellee.


This is Ed Silverman's report on the case.


Appeals court rules FDA ‘arbitrarily and capriciously’ approved a rare
disease drug
Ed Silverman
By Ed Silverman  Sept. 30, 2021

In a closely watched battle over regulatory decision-making, a federal
appeals court ruled the U.S. Food and Drug Administration wrongly approved
a rare disease medicine made by a small, family-run company because another
drug maker already held the exclusive right to market a similar treatment.

The lawsuit was filed by Catalyst Pharmaceuticals (CPRX), which accused the
agency of violating federal law two years ago when it unexpectedly approved
a medicine made by Jacobus Pharmaceuticals for treating children with a
rare neuromuscular disorder called Lambert-Eaton myasthenic syndrome, or
LEMS. The FDA had previously endorsed a Catalyst drug to treat LEMS, but
only for adults.


The unusual saga emerged from a race between the companies to be the first
to win FDA approval for a LEMS treatment. For 20 years, Jacobus distributed
its unapproved medicine for free to a few hundred patients in the U.S.
thanks to the compassionate use program. Meanwhile, Catalyst saw an
opportunity to mine a potentially lucrative market and licensed its drug
from another company.

By November 2018, Catalyst won the race when the FDA granted approval to
treat adults. But the company quickly encountered controversy when it
announced its medicine, called Firdapse, would cost $375,000 or more,
depending upon patient weight. Although Catalyst launched a patient
assistance program that lowered costs dramatically, health insurers were
left to pick up the tab.

A few months later, Sen. Bernie Sanders (I-Vt.) accused Catalyst of
“immoral exploitation” and later asked the FDA to refrain from taking any
regulatory actions that would prevent Jacobus from continuing to make its
drug available. Besides pricing, some LEMS patients were complaining that
the Catalyst drug was insufficiently effective.

In May 2019, the FDA made an unexpected move by approving the Jacobus drug,
called Ruzurgi, but only for children. However, the maneuver left the door
open for doctors to prescribe the Jacobus drug to any LEMS patient, since
physicians have the right to prescribe “off label,” a term that means a
doctor has decided a patient could benefit from a treatment even if it is
not approved for a particular use.

The move infuriated Catalyst, which quickly filed a lawsuit accusing the
FDA of illegally approving the Jacobus treatment in response to pressure
over the prescription drug pricing controversy. Catalyst argued the agency
applied different approval standards and unfairly trampled on its right to
seven years of exclusive marketing, a designation for so-called orphan
drugs that treat small numbers of patients.

During court proceedings, Catalyst sought to bolster its case by pointing
to internal FDA emails that mentioned the pricing controversy. The company
argued FDA staff inappropriately cited pricing to justify approval of the
Jacobus drug, since the FDA is not allowed to consider pricing when
reviewing medicines. And Catalyst insisted the FDA approval of the Jacobus
drug, which costs about half as much, should be nullified.

For its part, the FDA maintained that, while both drugs contain the same
active ingredient, LEMS in adults is not the same as LEMS in children (the
Ruzurgi approval covered ages 6 to 17 years old). Consequently, the Ruzurgi
approval for children did not violate the orphan drug exclusivity that was
awarded the Catalyst treatment for adults.

 [ snip]

James Love.  Knowledge Ecology International
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U.S. office phone +1.202.332.2670
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