This company develops therapeutics for rare diseases, focusing on clinical and regulatory strategies informed by key thought leaders, physicians, patients, and advocacy groups. They aim to bring new therapies to market by navigating complex development challenges with data-driven approaches.
Funding
$60M raised to dateRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.
Founders
Product
Problem
Many rare diseases lack effective treatments, leaving patients with limited or no therapeutic options. Traditional drug development faces challenges in addressing the unique complexities and small patient populations associated with these conditions. This results in significant unmet needs and a prolonged search for solutions for affected individuals.
Solution
Zevra Therapeutics is a biopharmaceutical company focused on acquiring, developing, and commercializing therapies for rare diseases. The company employs a data-driven approach, working closely with key opinion leaders, physicians, patients, and advocacy groups to navigate the complexities of rare disease drug development. Zevra's strategy involves identifying promising medicines, overcoming clinical and regulatory hurdles, and delivering treatments to patients with unmet needs. The company has a pipeline of product candidates targeting neurological and metabolic rare diseases, as well as rare sleep disorders.
Target Audience
The primary target audience includes patients and families affected by rare diseases, as well as physicians and healthcare providers specializing in the treatment of these conditions.
Features
- Focus on therapeutics for rare diseases with significant unmet needs.
- Data-driven approach to drug development and regulatory strategy.
- Collaboration with key opinion leaders, physicians, patients, and advocacy groups.
- Commercialized products include MIPLYFFA® (arimoclomol) for Niemann-Pick disease type C and OLPRUVA® (sodium phenylbutyrate) for certain urea cycle disorders.
- Investigational product KP1077, a prodrug of d-methylphenidate (d-MPH), for the treatment of idiopathic hypersomnia (IH) and narcolepsy.
- Celiprolol, a New Chemical Entity currently in Phase 3 development for the treatment of COL3A1-positive VEDS patients to potentially reduce the risk of arterial and other hollow organ clinical events.
- Orphan drug designation for KP1077 in Idiopathic Hypersomnia.
- Clinical trials are conducted to evaluate the safety and efficacy of product candidates.