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Orphan Exclusivity In Catalyst’s Aftermath: FDA Inaction Leaves Rare Disease Sponsors In Limbo – Food and Drugs Law

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More than a year has passed since the US Court of Appeals for
the Eleventh Circuit’s decision in Catalyst Pharms., Inc.
v. Becerra
struck down the US Food and Drug
Administration’s (FDA) indication-specific interpretation of
orphan drug exclusivity (ODE).1 In response to that
September 2021 decision, FDA’s Office of Orphan Products
Development (OOPD) has deferred all of its pending orphan
exclusivity determinations. In fact, OOPD has not
publicly recognized orphan exclusivity for any product approved
since November 2021
. Rather than recognize a broad,
disease-specific scope of orphan exclusivity consistent with the
Eleventh Circuit’s opinion, the agency appears to be waiting
for a legislative fix to effectively overrule the Catalyst
decision. The status of that legislative fix is in jeopardy,
however, in light of the recent passage2 of a
“clean” version of the FDA user fee reauthorization
provisions—without any changes to the Orphan Drug Act. As a
result, sponsors remain in the dark about the impact of the court
decision, and FDA has given little indication how the agency
intends to proceed.

The Catalyst Decision

Briefly, the Catalyst decision held that FDA’s
indication-specific interpretation of the scope of ODE was
incompatible with the Orphan Drug Act’s exclusivity provision,
which describes a “rare disease or condition.” Under
FDA’s historical approach, which had been codified in agency
regulations since 2013, a drug product approved for an
“indication or use” that is narrower than the “rare
disease or condition” for which the orphan designation was
granted would obtain exclusivity only for the approved indication
or use. However, the Eleventh Circuit held in Catalyst
that the scope of exclusivity was as broad as the rare disease or
condition for which designation was granted, even if a drug
product’s approval was for a narrower indication. In the
context of the Catalyst case, the court thus concluded
that the orphan exclusivity for Catalyst’s Firdapse
(amifampridine phosphate) blocked approval of Jacobus
Pharmaceutical Co.’s (Jacobus’s) competitor product,
Ruzurgi (amifampridine), for a different
indication.3

The Aftermath, Part 1: Catalyst and Jacobus Settle Their
Dispute, Leaving Firdapse the Sole Product on the Market

Following the Eleventh Circuit decision, intervenor-defendant
Jacobus initially submitted a petition for a writ of certiorari to
the Supreme Court in hopes of overturning the appellate court’s
decision. FDA did not join or otherwise seek further review. Before
the Supreme Court ruled on the Jacobus petition, however, the issue
became moot—at least with respect to the two amifampridine
products. In the context of related patent litigation brought by
Catalyst against Jacobus, the two companies announced a settlement
in July 2022, whereby Jacobus agreed to withdraw its petition to
the Supreme Court and Catalyst dismissed its patent infringement
claims and acquired rights to distribute Jacobus’s Ruzurgi in
the US.4

What is more, in late September 2022, FDA approved a supplement
to the Firdapse NDA for use in pediatric patients older than 6
years of age. As a result, the outcome that FDA appears to have
sought to avoid—a single approved amifampridine product on
the market—remains the status quo, at least until the
expiration of the seven-year orphan exclusivity period for
Catalyst’s Firdapse in November 2025.

The Aftermath, Part II: What About All the Other Orphan Drug
Products?

The impact of the Catalyst decision on FDA and the rare
disease industry has been significant. As noted in our previous Legal Update, the scope of orphan exclusivity
for existing products remains unclear. For products approved since
the Catalyst decision, the situation is even worse. These
sponsors cannot even be certain that their orphan-designated
products will be protected by orphan exclusivity, because FDA’s
OOPD has effectively stopped making exclusivity determinations
since late 2021. The agency appears to be seeking to avoid having
to recognize an orphan exclusivity broader than a product’s
approved use, as the Catalyst decision would seem to
require of FDA.

Key to the agency’s inaction had been the
possibility—or even expectation—that the user fee
reauthorization legislation would include a provision effectively
overruling Catalyst and codifying FDA’s orphan
exclusivity regulation. Indeed, the user fee bills initially
released by both the House and the Senate contained provisions
amending the statutory exclusivity provision to narrow the scope of
ODE to the “approved use or indication.”5 Both
bills also included language designed to have the new provisions
applied retroactively:

(b) Application of Amendments. The amendments made by subsection
(a) shall apply with respect to any drug designated under section
526 of the Federal Food, Drug, and Cosmetic Act,
regardless of the date on which the drug was so
designated, and regardless of the date on which the drug was
approved
under section 505 of such Act or licensed
under section 351 of the Public Health Service Act.6

Unfortunately for FDA, those proposed amendments were dropped
from the “clean” user fee reauthorization provisions
included in the legislation passed in late September. Amidst
reports that both houses of Congress will in the coming months
revisit proposals dropped from the user fee bills, FDA appears
poised to continue waiting on Congress to resolve the agency’s
conundrum.

So where does that leave rare disease sponsors with
investigational or approved drug products? In limbo, unfortunately.
Given OOPD’s inaction, there are (as of the date of
publication) more than 60 products approved for more than 70
orphan-designated indications for which OOPD has issued
no public orphan exclusivity determination
. For more
than a year, no “ODE” codes have been added to the Orange
Book, and no new listings of orphan exclusivity appear in
OOPD’s searchable orphan designation database.

OOPD has made one concession to sponsors: the OOPD database now
includes a “TBD” notation for those products whose
eligibility for orphan drug exclusivity has not been determined.
(However, the “TBD” notation only appears when query
results are listed in the “condensed” format but not if
results are listed in the “detailed” format or in an
Excel file.)

Our Take, for FDA

We urge FDA to take a more nuanced approach. For products
approved since the Catalyst decision was finalized, OOPD
should make a final determination about whether the product has
earned ODE and make that determination public by noting the
exclusivity in the Orange Book (with an “ODE” code) and
by listing a date in the Exclusivity End Date field in the orphan
designation database, i.e., the date that is seven years after the
relevant approval. Doing so would provide sponsors greater clarity,
without forcing the agency to address the scope of the ODE unless
and until absolutely necessary—or the above-noted legislative
fix arrives. In the meantime, sponsors are left in limbo, without
the ability to provide updates to the public and the investment
community about the exclusivity for their orphan-designated
products.

The agency could also consider opening a public docket to
solicit stakeholder feedback on proposed resolutions, should a
legislative fix not arrive soon.

Our Take, for Sponsors

For sponsors of recently approved orphan products, who are
unlikely to face competitor applications in the short term, further
patience will likely be required as the agency waits to see whether
the legislative fix will arrive. In the meantime, we recommend that
sponsors carefully assess whether a competitor is likely to seek
approval for the indication or use whose orphan exclusivity has not
yet been recognized by FDA. In that situation, consideration should
be given to a more assertive approach, including petitioning FDA to
award the orphan exclusivity or preparing for potential litigation
prior to a competitor approval.

Footnotes

1. Catalyst Pharms., Inc. v. Becerra, 14 F.4th
1299 (11th Cir. 2021).

2. H.R. 6833, “The Continuing Appropriations and
Ukraine Supplemental Appropriations Act, 2023” Sept. 30,
2022.

3. FDA initially granted final approval to Jacobus for
“treatment of Lambert-Eaton myasthenic syndrome (LEMS) in
pediatric patients 6 to less than 17 years of age” on the
basis that Firdapse’s ODE was limited to its approved use,
“treatment of Lambert-Eaton myasthenic syndrome (LEMS) in
adults.”

4. See Catalyst Press Release, Catalyst
Pharmaceuticals Announces Settlement of U.S. Patent Litigation and
Resolution of Litigation Challenging Ruzurgi® Approval with
Jacobus Pharmaceutical
, (July 12, 2022), available at
https://ir.catalystpharma.com/news-releases/news-release-details/catalyst-pharmaceuticals-announces-settlement-us-patent.

5. See, e.g., § 510(a), S. 4348,
117th Congress, 2d session (July 13, 2022).

6. Id. at § 510(b) (emphases added)
(internal citations omitted).

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