==ARS Pharmaceuticals (SPRY) : FDA Declines to Approve No-Shot Alternative to EpiPen, Asks for Additional Data

 

ARS Pharmaceuticals receives Complete Response Letter from FDA for neffy NDA

• Co announced that the FDA issued a Complete Response Letter (CRL) regarding its New Drug Application (NDA) for neffy (epinephrine nasal spray) in the treatment of Allergic Reactions (Type I), including anaphylaxis for adults and children =30 kg. ARS Pharma plans to submit a Formal Dispute Resolution Request (FDRR) to appeal the issuance of this CRL.
• In the letter, the FDA requested completion of a pharmacokinetic/pharmacodynamic study assessing repeat doses of neffy compared to repeat doses of an epinephrine injection product under allergen-induced allergic rhinitis conditions to support approval. This request comes after the recommendation of the FDA Advisory Committee (PADAC) in May 2023 to approve neffy without the need for additional studies to demonstrate its efficacy or safety. Further, FDA and ARS Pharma previously aligned in August 2023 on final physician's labeling and a post-marketing requirement to conduct this study as informative for labeling.
  • The PADAC meeting was held on May 11, 2023, and concluded a favorable benefit-risk profile of neffy, with a 16:6 vote in favor for adults and 17:5 vote in favor for children (=30 kg) for the treatment of patients with allergic reactions (Type I), including anaphylaxis. In that session, no member of the Committee raised specific concerns about the result of the completed study in people with allergen-induced acute rhinitis with single-dose neffy, which showed enhanced absorption during the time period when a clinical response would be expected.
• As ARS Pharma previously agreed with FDA to conduct a repeat-dose study under allergen-induced allergic rhinitis conditions as a post-marketing commitment, ARS Pharma anticipates a resubmission to the FDA in the first half of 2024, positioning ARS Pharma for an anticipated FDA action date in the second half of 2024.
• ARS Pharma expects to have anticipated cash, cash equivalents and short-term investments on hand of approximately $195 million at the time of the anticipated launch of neffy, if approved in the second half of 2024.
• The CRL requested additional information on nitrosamine impurities to be tested for based on new draft guidance issued after the neffy NDA submission. ARS Pharma does not believe the additional testing would be a rate-limiting step for its resubmission to the FDA.

Mannkind (MNKD): FDA declines to approve lung drug

 

 

 

Mannkind provides up on Tyvaso DPI NDA

• MannKind Corporation has learned that the FDA issued a complete response to United Therapeutics (UTHR) regarding the New Drug Application (NDA) for Tyvaso DPI for the treatment of pulmonary arterial hypertension and pulmonary hypertension associated with interstitial lung disease.
• The FDA declined to approve the NDA at this time, noting only one deficiency related to an open inspection issue at a third-party analytical testing center for treprostinil drug substance, the active ingredient of Tyvaso DPI. The complete response did not pertain to MannKind, and no issues were cited by the FDA as it relates to MannKind's facility in Connecticut for manufacturing, testing and packaging of finished Tyvaso DPI, including its associated device.

-=Zogenix (ZGNX) receives refusal to file letter for its seizure drug

Zogenix receives Refusal to File Letter from FDA for FINTEPLA New Drug Application treating seizures associated with Dravet syndrome

Co received a Refusal to File (RTF) letter from the FDA regarding its New Drug Application (NDA) for FINTEPLA (ZX008, fenfluramine hydrochloride) for the treatment of seizures associated with Dravet syndrome.

Upon its preliminary review, the FDA determined that the NDA, submitted on February 5, 2019, was not sufficiently complete to permit a substantive review. In the letter, the FDA cited two reasons for the RTF decision: first, certain non-clinical studies were not submitted to allow assessment of the chronic administration of fenfluramine; and, second, the application contained an incorrect version of a clinical dataset, which prevented the completion of the review process that is necessary to support the filing of the NDA.

The FDA has not requested or recommended additional clinical efficacy or safety studies.

The Company will seek immediate guidance, including a Type A meeting with the FDA, to clarify and respond to the issues identified in the RTF letter.

Zogenix's Marketing Authorization Application (MAA) for FINTEPLA for the treatment of seizures associated with Dravet syndrome was previously accepted for review by the European Medicines Agency (EMA), and the Company anticipates an approvability decision could be reached by the EMA in the first quarter of 2020.

=Akcea Therapeutics (AKCA) and Ionis (IONS) : FDA rejects rare disease drug

The FDA rejected a drug developed by Massachusetts-based biotech Akcea Therapeutics Inc. in partnership with California-based Ionis Pharmaceuticals that's intended to treat a rare lipid disorder, halting hopes for a treatment for the currently unserved, debilitating disease.


Akcea Therapeutics and Ionis (IONS) receive Complete Response Letter for WAYLIVRA from FDA  

• Akcea Tx (AKCA) an affiliate of Ionis Pharma (IONS) received a Complete Response Letter (CRL) from the Division of Metabolism and Endocrinology Products of the U.S. FDA regarding the New Drug Application (NDA) for WAYLIVRA (volanesorsen).
• "We are extremely disappointed with the FDA's decision. FCS is an ultra-rare and debilitating disease. Our disappointment extends to the patient and physician community who currently do not have a treatment available to them. We continue to feel strongly that WAYLIVRA demonstrates a favorable benefit/risk profile in people with FCS as was reflected in the positive outcome from our Advisory Committee hearing in May. We will continue to work with the FDA to confirm the path forward,"

Valeant Pharmaceuticals (VRX) : psoriasis treatment rejection by FDA

• Note: Valeant Pharma (VRX) has been renamed Bausch Health (NYSE:BHC)  (July 2018)

 

 Valeant Pharma receives Complete Response Letter (CRL) regarding the New Drug Application for DUOBRII (IDP-118) lotion in the treatment of plaque psoriasis
"The CRL did not specify any deficiencies related to the clinical efficacy or safety of DUOBRII and no issues with CMC* processes. The CRL only noted questions regarding pharmacokinetic data," said Joseph C. Papa, chairman and CEO, Valeant. "We are working to resolve this matter expeditiously and have already requested a meeting with the FDA. We hope to bring forward this important new treatment option for those who suffer from plaque psoriasis as quickly as possible."

-=Alkermes (ALKS) : refusal to file letter from FDA

Alkermes receives refusal to file letter from FDA for ALKS 5461  

• Co announced that it received a Refusal to File letter from the FDA regarding its New Drug Application for ALKS 5461, a once-daily, oral investigational medicine with a novel mechanism of action for the adjunctive treatment of major depressive disorder in patients with an inadequate response to standard antidepressant therapies.
• Upon its preliminary review, the FDA has taken the position that it is unable to complete a substantive review of the regulatory package, based on insufficient evidence of overall effectiveness for the proposed indication, and that additional well-controlled clinical trials are needed prior to the resubmission of the NDA for ALKS 5461. In addition, FDA has requested the conduct of a bioavailability study to generate additional bridging data between ALKS 5461 and the reference listed drug, buprenorphine.

=Acorda Therapeutics (ACOR) : FDA rejects application for Parkinson's treatment

Acorda Therapeutics received a Refusal to File letter from the FDA regarding its NDA for INBRIJA; co will seek immediate guidance, including a Type A meeting with the FDA, to respond to the issues

• Upon its preliminary review, FDA determined that the NDA, submitted on June 26, 2017, was not sufficiently complete to permit a substantive review. FDA specified two reasons for the RTF: first, the date when the manufacturing site would be ready for inspection, and, second, a question regarding the submission of the drug master production record. FDA also requested additional information at resubmission, which was not part of the basis for the RTF.
• The Company will seek immediate guidance, including a Type A meeting with the FDA, to respond to the issues, which it believes are addressable, and to seek clarification of what additional information will be required. The FDA has not requested or recommended additional clinical efficacy or safety studies.