Strength

1. World leader in developing and commercializing innovative biopharmaceuticals for rare diseases driven by genetic causes: BioMarin is well renowned for rendering innovative therapeutics to patients with life-threatening conditions. With seven products in its portfolio and a fully-integrated multinational organization in place, there is whetted focus on developing flagship best-of-the-breed therapeutics that provide meaningful advances to patients who live with serious and life-threatening rare genetic diseases. This target patient’s coup primarily comprises of children, suffering from diseases so rare, that the entire patient population can number as few as 1,000 people worldwide. These conditions are often inherited, difficult to diagnose, progressively debilitating, have few, if any, treatment options, and are usually ignored. BioMarin is also the world leader in metabolic disease innovation with multiple products commercialized and a growing pipeline of product candidates to address non-diagnosed medical needs.

2. Bringing speedy innovative therapies to meet critical patient needs: Time is very crucial to patients with rare diseases, and Biomarin has therapies for them. The efficiency and speed of BioMarin’s research, development, manufacturing, and commercial efforts works at a commendable pace to exigently deliver therapies. This American biotechnology firm’s track record of developing and commercializing new treatments has been substantially faster than the industry average. CenterWatch, a leading source for global clinical trial information, has coronated BioMarin as one of the fastest drug developers in the industry. BioMarin also ranked tenth on Forbes list of innovative companies in 2015 and fourth best mid-size employer in America in 2019.

3. Diversified rare disease player: By and large mid-sized rare disease pharmaceuticals have heavy dependency on a single drug or therapeutic space. BioMarin Pharmaceutical, however, stands as an exception with multiple gems in its portfolio. The company's enzyme replacement therapy for genetic disease Morquio A, Vimizim, accounts for almost one-third of its total revenues. This drug generated $125.8 million revenues in the first quarter and is expected to rake up an additional $530-570 million in revenue, close to 30% of the company’s fiscal 2019 revenue guidance of $1.68-1.75 billion. BioMarin also has Naglazyme, the first FDA-approved enzyme replacement therapy for a rare genetic disease, MPS VI (mucopolysaccharidosis type VI), which is projected to attract $350-380 million in fiscal 2019. Aldurazyme, the only FDA-approved treatment for MPS I, and Brineura, the only enzyme replacement therapy approved for treating CLN2 disease, are some other shining jewels in BioMarin’s basket.

4. Bellwether in several therapeutic treatments for life-threatening conditions: BioMarin's core business and research is in enzyme replacement therapies (ERTs). This American multinational biotechnology company was the first company to provide therapeutics for mucopolysaccharidosis type I (MPS I) an inherited, often life-threatening lysosomal disorder caused by a deficiency of the lysosomal enzyme, alpha-L-iduronidase., by manufacturing laronidase (Aldurazyme, commercialized by Genzyme Corporation). Besides being the flagship and only approved therapy for treating MPS1 (both by FDA and European Commission in 2003), it has also been designated orphan drug status in both the United States and the European Union and received approvals in numerous countries across the globe. BioMarin was also aced the production and commercialization of therapeutics for phenylketonuria (PKU). PALYNZIQ® (pegvaliase-pqpz) Injection is the first FDA (Food and Drug Administration) and EMA (European Medicines Agency)-approved (in 2018) enzyme substitution therapy designed to address the underlying cause of phenylketonuria (PKU), a rare and genetic brain-threatening condition marked by an inability to break down phenylalanine (Phe). Brineura from BioMarins’s stable is again the first approved therapy for any form of Batten disease and the recipient of an orphan drug status in the United States and the European Union. BioMarin’s Firdapse, is the flagship specific approved therapy in the European Union for the treatment of Lambert-Eaton Myasthenic Syndrome (LEMS) and designated as an orphan drug internationally. In 2019-2020, it is purportedly going to revolutionized the Haemophilia A space.

Weakness

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Opportunity

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Threat

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