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Catalyst Pharmaceuticals Comments on FDA s Announcement of its Position on Orphan Drug Exclusivity In Light of the 11 th Circuit Decision in Catalyst Pharmaceuticals, Inc. v. Becerra Announced Earlier Today- Notice Recei

Key Takeaway: Catalyst Pharmaceuticals has commented on a recent FDA announcement regarding orphan drug exclusivity following a decision by the 11th Circuit Court. The FDA confirmed that it will comply with the court ruling favoring Catalyst related to its drug FIRDAPSE, while still applying regulations on orphan drug exclusivity for other medications. Additionally, Catalyst noted that it acquired rights to RUZURGI and has previously expanded its FIRDAPSE label to include children aged 6-17. However, a recent application from Teva Pharmaceuticals seeks authorization for a generic version of FIRDAPSE, prompting Catalyst to prepare to enforce its intellectual property rights.

Market Sentiment Analysis

POSITIVE FACTORS

  • FDA's announcement confirms the previous decision in favor of Catalyst.
  • Catalyst has successfully expanded FIRDAPSE's label for younger patients.
  • The company plans to vigorously defend its intellectual property regarding FIRDAPSE.

CONCERNS & RISKS

  • Teva Pharmaceuticals has filed an abbreviated new drug application for a generic version of FIRDAPSE.
  • The FDA's position could impact future orphan drug exclusivity interpretations.

Full Press Release Details

Catalyst Pharmaceuticals Comments on FDA s Announcement of its Position on Orphan Drug Exclusivity In Light of the 11th Circuit Decision in Catalyst Pharmaceuticals, Inc. v. Becerra
Announced Earlier Today-Notice Received from ANDA Filer for FIRDAPSE
CORAL GABLES, Fla., Jan. 23, 2023 Catalyst Pharmaceuticals, Inc. ( Catalyst )
(NASDAQ: CPRX), a commercial-stage biopharmaceutical company focused on in-licensing, developing, and commercializing novel medicines for patients living with rare diseases, today commented on the FDA s
notice setting forth its position on Orphan Drug Exclusivity in light of the 11th Circuit s decision in Catalyst Pharmaceuticals, Inc. v. Becerra. The FDA announcement states that
while the FDA is complying with the 11th Circuit decision in Catalyst s favor with respect to FIRDAPSE , the FDA intends to continue
to apply its regulations tying the scope of orphan drug exclusivity to the uses or indications for which a drug is approved with respect to other orphan drugs. Catalyst reports that it is not affected by the FDA s newly announced position and
that the FDA s announcement confirms the FDA s previous decision to set aside the approval of RUZURGI as a result of the 11th
Circuit s decision. Further, Catalyst acquired the United States rights to RUZURGI , including all intellectual property relating thereto, in July 2022. Finally, Catalyst s
supplemental New Drug Application expanding its label for FIRDAPSE to children ages 6-17 was approved by the FDA in September 2022.
Separately, Catalyst announced this morning that an abbreviated new drug application has been filed by Teva Pharmaceuticals, Inc. seeking authorization from
the FDA to manufacture, use, or sell a generic version of FIRDAPSE in the United States. As Catalyst has previously reported, the filing of a Paragraph IV challenge to FIRDAPSE was not unexpected, and Catalyst intends to vigorously enforce its intellectual property rights relating to FIRDAPSE .
About Catalyst Pharmaceuticals
Pharmaceuticals is a commercial-stage biopharmaceutical company focused on in-licensing, developing, and commercializing novel medicines for patients living with rare diseases. With exceptional patient
focus, Catalyst is committed to developing a robust pipeline of cutting-edge, best-in-class medicines for rare diseases. Catalyst s New Drug Application for
FIRDAPSE (amifampridine) Tablets 10 mg for the treatment of adults with Lambert-Eaton myasthenic syndrome ( LEMS ) was approved in 2018 by the U.S. Food & Drug Administration and FIRDAPSE is commercially
available in the United States as a treatment for adults and children ages six to seventeen with LEMS. Further, Canada s national healthcare regulatory agency, Health Canada, has approved the use of FIRDAPSE for
the treatment of adult patients in Canada with LEMS.
For more information, visit Catalyst s website at www.catalystpharma.com.
Forward-Looking Statements
This press release contains
forward-looking statements. Forward-looking statements involve known and unknown risks and uncertainties, which may cause Catalyst s actual results in future periods to differ materially from forecasted results. A number of factors, including
those set forth in Catalyst s Annual Report on Form 10-K for the fiscal year 2021, its Quarterly Report on Form 10-Q, and its other filings with the U.S.
Securities and Exchange Commission ( SEC ), could adversely affect Catalyst. Copies of Catalyst s filings with the SEC are available from the SEC, may be found on Catalyst s website, or may be obtained upon
request from Catalyst. Catalyst does not undertake any obligation to update the information contained herein, which speaks only as of this date.

Frequently Asked Questions

What is the recent FDA announcement about Catalyst Pharmaceuticals?

The FDA is complying with the 11th Circuit decision favoring Catalyst but will apply orphan drug exclusivity regulations to other drugs.

How does the FDA's decision affect RUZURGI?

The FDA's announcement confirms the set aside of RUZURGI's approval following the 11th Circuit's ruling.

What is the significance of FIRDAPSE for Catalyst Pharmaceuticals?

FIRDAPSE is Catalyst's approved treatment for Lambert-Eaton myasthenic syndrome in both adults and children.

Who has filed an application regarding FIRDAPSE?

Teva Pharmaceuticals has filed an abbreviated new drug application for a generic version of FIRDAPSE.

When was FIRDAPSE approved for children ages 6-17?

FIRDAPSE's label expansion for children aged 6-17 was approved by the FDA in September 2022.

Last updated: Jan 23, 2023