June 2022 decisions expected from the FDA

At Prime Therapeutics (Prime), we’ve positioned ourselves to best prepare our clients to manage new drugs. Our clinical and trade relations teams keep a keen eye on drugs likely to be approved by the U.S. Food and Drug Administration (FDA).

Drug pipeline for June 2022:

6/5/2022: sodium phenylbutyrate
The FDA is reviewing Acer Therapeutics and Relief Therapeutics’ sodium phenylbutyrate for the treatment of urea cycle disorders (UCDs). UCDs are a group of disorders caused by genetic mutations that result in a deficiency in enzymes that catalyze the urea cycle. Enzyme deficiencies in the urea cycle can lead to an excess accumulation of ammonia in the bloodstream resulting in fatigue, headaches, multi-organ failure, and neurologic disorders.¹ Sodium phenylbutyrate is seeking approval via the 505(b)(2) pathway using Horizon Therapeutics’ Buphenyl (sodium phenylbutyrate) as its reference product.² Current treatment options for patients with UCDs include Horizon Therapeutics’ Ravicti^® (glycerol phenylbutyrate) and Buphenyl^® (sodium phenylbutyrate).^1,2 Acer Therapeutics and Relief Therapeutics’ sodium phenylbutyrate is designed to improve palatability, a common cause of noncompliance, through its taste-masked formulation that lacks bitterness and dissolves quickly in the stomach.²

6/27/2022: tebipenem pivoxil hydrobromide
The FDA is reviewing Spero Therapeutics’ tebipenem pivoxil hydrobromide (tebipenem HBr) for the treatment of complicated urinary tract infections (cUTI), including acute pyelonephritis (AP). If approved, tebipenem HBr will be the first broad-spectrum oral extended release carbapenem to treat bacterial infections like cUTI and AP.³ The NDA submission is supported by results of a pivotal Phase 3 trial, ADAPT-PO, that tested oral tebipenem HBr against an intravenous (IV) regimen. Results showed that oral tebipenem HBr demonstrated non-inferiority to IV ertapenem in cUTI and AP.³ The FDA has granted Priority Review for tebipenem HBr for the treatment of cUTI, including AP.³ Similar products include generic IV carbapenems.

6/2022: spesolimab
Boehringer Ingelheim’s spesolimab is seeking FDA approval for the treatment of flares in generalized pustular psoriasis (GPP). GPP is a rare, life-threatening neutrophilic skin disease, which is distinct from plaque psoriasis.⁴ It is characterized by episodes of widespread eruptions of painful, sterile pustules on the skin.⁴ Flares greatly affect a person’s quality of life and can lead to hospitalization with serious complications, including heart failure, renal failure, sepsis, and death. Spesolimab is seeking approval based on results of the pivotal Phase 2 Effisayil™ 1 trial, which showed that spesolimab significantly improved signs and symptoms of GPP in patients experiencing a flare when compared to placebo.⁵ The FDA has granted Priority Review for spesolimab for the treatment of GPP flares.⁴ Currently, there are no approved treatment options in the U.S. for GPP flares, however, therapies like oral cyclosporine, intravenous infliximab, and IL-17 and IL-23 inhibitors have commonly been used off-label.⁶

While the information in this newsletter is from sources we believe to be reliable, we do not warrant that the information in this document is free from error. Use it only as a guide. Statements regarding drugs or manufacturers are not intended as promotion; those statements should not be used to make assumptions about formulary status. Each trademarked drug name is the property of its respective owner.

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References

1. https://www.biospace.com/article/releases/relief-announces-that-collaboration-partner-acer-therapeutics-was-issued-u-s-patent-11-202-767-covering-acer-001-methods-of-use-for-treatment-of-urea-cycle-disorders-and-maple-syrup-urine-disease/
2. https://www.fiercepharma.com/drug-delivery/acer-prepares-to-file-for-fda-approval-taste-masked-formulation-to-challenge-horizon
3. https://www.globenewswire.com/news-release/2022/04/06/2418056/0/en/Spero-Therapeutics-Tebipenem-Pivoxil-Hydrobromide-Phase-3-Data-Published-in-The-New-England-Journal-of-Medicine.html#:~:text=Tebipenem%20HBr%20is%20an%20investigational,have%20limited%20oral%20treatment%20options.
4. https://www.boehringer-ingelheim.us/press-release/us-fda-grants-priority-review-spesolimab-treatment-flares-patients-generalized
5. https://www.nejm.org/doi/full/10.1056/NEJMoa2111563
6. https://www.uptodate.com/contents/pustular-psoriasis-management?search=treatment%20of%20GPP%20flares&source=search_result&selectedTitle=1~150&usage_type=default&display_rank=1#H167785525