Genzyme is a leading biotechnology firm focused primarily on developing drugs for rare diseases and chronic illnesses.Since 1981, the company has grown from a small start-up to a diversified business with more than 12,000 employees in locations spanning the globe and 2009 revenues of $4.5 billion. The company's products and services are focused on rare inherited disorders, kidney disease, orthopaedics, cancer, transplant and immune disease, and diagnostic testing.
Genzyme’s continued success relies on its ability to continue to dominate specialized, rare disease markets that have small patient populations, as well as its ability to expand market share for existing drugs. Genzyme’s recent acquisition spree has brought it a number of new products that exhibit growth potential, but its market share must continue to grow steadily to allow for Genzyme’s continued success. Genzyme faces similar obstacles to most other biotechnology firms, including concentrated risk in a small number of products, FDA regulation and approval, Medicare reimbursement levels, and litigation. Genzyme has a diverse product pipeline with twenty drugs in clinical trials and the company’s long-term success depends on their ability to develop effective drugs, get FDA approval for these drugs, and bring these drugs to market. Additionally, any changes to Medicare reimbursement plan could adversely affect Genzyme’s revenue if clinics and doctors become less willing to use Genzyme’s products due to reduced reimbursement rates.
Genzyme, Inc. (NASDAQ: GENZ) is a leading biotechnology firm that focuses on developing treatments for rare diseases and chronic illnesses. The company’s products are categorized in the following four categories:
First Quarter 2010 Summary
Genzyme reported revenues of $1.07 billion, compared to $1.15 billion in the same period last year. The company reported a net loss for the quarter of $114.9 million, compared to net income of $195.5 million in the first quarter of 2009. Genzyme's decline in revenue is the result of limited shipments of Cerezyme and Fabrazyme due to product supply constraints.The sharp decline in net income is the result of a $175 million expense associated with the consent decree for the company’s Allston manufacturing facility. Genzyme received a draft consent decree from the FDA regarding its Allston manufacturing plant. The draft provides for an up-front disgorgement of past profits of $175 million.
Within the Personalized Genetic Health segment (previously called Genetic Diseases) first-quarter sales of Myozyme increased 28 percent to $86.1 million from $67.4 million in the same period in 2009. First-quarter revenue was down from fourth-quarter 2009 sales, reflecting an $8 million shipment that was delayed for a release hold at the end of the first quarter.
New patients continued to begin treatment with Myozyme during the first quarter. Expansion is ongoing worldwide, and Genzyme plans new regulatory filings in more than 15 countries during 2010.
Genzyme in December reopened enrollment in the Alglucosidase Alfa Temporary Access Program (ATAP), a U.S. program which provides access to treatment for severely affected adults with Pompe disease prior to commercial approval of Lumizyme. The ATAP program will remain active until commercial approval of Lumizyme. Genzyme is currently providing therapy free of charge to more than 200 patients in the United States.
First-quarter sales of Cerezyme were $179.1 million compared with $296.0 million in the first quarter of last year. Cerezyme revenue increased 70 percent from $105.4 million in the fourth quarter of 2009, reflecting the resumption of product shipments to patients in approximately 100 countries. Sales of Fabrazyme in the first quarter were $53.2 million, compared with $122.2 million in the same period last year.
Within the Biosurgery segment, sales of Synvisc increased 26 percent to $79.5 million from $63.2 million in the first quarter of 2009. This growth was driven by Synvisc-One, which was launched in the United States in March 2009 and is the only single-injection viscosupplement approved for the treatment of osteoarthritis knee pain. First-quarter 2010 sales were down from $95.4 million in the fourth quarter of 2009, reflecting the annual seasonality trend in the viscosupplement market, in which sales typically decline in the first quarter, and peak during the second quarter.
Sales of Sepra products grew 8 percent to $37.2 million, from $34.3 million in the same period last year, with growth driven by increasing use of the Seprafilm adhesion barrier in C-section and other gynecologic procedures.
Within this segment, sales of Genzyme’s sevelamer therapies, Renvela and Renagel, were $164.6 million, compared with $170.6 million during the first quarter of 2009. While sevelamer volume grew by 4 percent year over year, Renagel pricing in Brazil and conversion to Renvela in the United States resulted in a decrease in revenue. Genzyme last year lowered the price of Renagel in Brazil to compete with generics marketed in that country. Genzyme had raised the price of Renagel in the United States last year to encourage conversion to Renvela, and revenue reflects the increasing percentage of Renvela sales within total U.S. sevelamer revenue. The U.S. market share for sevelamer grew during the first quarter and is currently 51 percent.
The ongoing launch of Renvela in Europe continues to progress well, with launches in the key markets of France, Spain, Italy and the U.K. planned in the coming months. The U.S. launch of the powder formulation of Renvela is also exceeding expectations.
Sales of Thyrogen increased 18 percent to $45.6 million from $38.8 million in the first quarter of 2009, driven by increased utilization of the treatment in thyroid remnant ablation procedures.
Within this segment, revenue from Mozobil grew 75 percent to $18.9 million from $10.8 million in the first quarter last year. Mozobil was launched in the United States in January 2009 and in Europe in August 2009, and additional worldwide launches are ongoing. First-quarter growth in this segment was also driven by sales of Clolar, which increased 36 percent to $24.7 million from $18.2 million in the same period a year ago, with significant volume growth seen in all major markets.
Sales of Thymoglobulin, were $52.9 million, compared with $50.7 million in the first quarter of 2009, due primarily to increased sales in Asia.
Results from several clinical trials have been submitted for presentation at the June American Society of Clinical Oncology meeting, including results of a phase 3 trial of Leukine in melanoma, Leukine data in prostate cancer, and Clolar data in myelodysplastic syndromes, non-Hodgkins lymphoma, and acute myeloid leukemia.
Genzyme’s risks are similar to those encountered by most biotechnology firms: majority of revenue concentrated in one or two main products, FDA approval, medical litigation, and over-aggressive acquisition and integration of other companies.
Genzyme faces $175 million in fines imposed by the FDA due to its unsatisfactory manufacturing facilities. The company came under fire from the FDA when an inspection of an Allston, Massachusetts, facility from Oct. 8, 2009 to Nov. 13, 2009 found quality control lacking, as many medically necessary products were found contaminated by metal, rubber, and glass particles as well as a viral contamination via a bioreactor which produced bulk drug quantities. The inadequate conditions of facilities also resulted in drug shortages -- which were compounded when Genzyme temporarily suspended manufacturing due to the quality control issues. As of May 2010, Genyzme was shipping its flagship product Cerezyme at 50% of demand and its product Fabrazyme at 30% of demand. Genzyme manufactures the Gaucher's disease drug imiglucerase (Cerezyme), the Fabry disease drug agalsidase beta (Fabrazyme), the thyroid cancer diagnostic thyrotropin alfa (Thyrogen), and the Pompe disease drug alglucosidase alfa (Myozyme) in the affected facility. Genzyme is the only supplier of a number of these enzyme replacement drugs. A consent decree signed with the FDA places Genzyme under a strict timetable in which to shape up its manufacturing facility.
As the sole provider of several drugs for rare genetic diseases, Genzyme is under intense pressure to maintain high quality control standards and maintain a steady supply of drugs to its patients.
Genzyme has an impressive stable of diverse products, but together, Cerezyme and Renagel account for more than 50% of the company’s revenue. Because of the small patient pool and possible alternative treatments, both of these drugs are vulnerable to competition, however their proven efficacy makes them more stable fixtures and they will prove difficult to displace unless a competing treatment is discovered to be significantly more effective.
As with all medicines in the United States, Genzyme’s products are subject to FDA approval and regulation. FDA approval requires tremendous research and at least three large clinical trials that prove the drug’s safety and success rates. Because Genzyme’s products are used across the globe, its drugs are also subject to the regulatory and approval processes of other countries’ drug regulation departments.
===Medicare Reimbursement=== cenira wuz here Because most of the patients who use Genzyme's treatments are eligible for Medicare, Genzyme's revenue is vulnerable to changes in Medicare reimbursement rates. Any reimbursement rate change would have a direct impact on Genzyme's revenue. Indirectly, rate changes may also cause doctors and clinics to be less willing to use Genzyme's products at the current market price for these products.
Despite the FDA’s stringent regulations, there is always the risk of unforeseen side effects that don’t become apparent until after the drug has come to market. Depending on the severity of the side effects, affected patients could potentially pursue medical litigation against Genzyme.
Since May 2004, Genzyme has acquired nine other specialized biotechnology firms that have greatly contributed to the Genzyme product pipeline. The risk of an aggressive acquisition policy is that Genzyme may fail to optimize new profit opportunities and lose focus on its core products
Because of Genzyme’s diverse product pipeline, multiple companies compete with Genzyme. The landscape of competitors shifts quickly due to constant innovations and discoveries in biotechnology. Shire PLC and Amicus Therapeutics are Genzyme’s primary competitors in rare disease markets. Shire has products to treat Fabry's Disease and Gaucher's Disease, two of Genzyme’s key markets. While neither of Shire’s drugs are currently available in the United States and would need to undergo head-to-head trials with Genzyme’s products before coming to market, Shire's drugs are potential competitors because of the small patient pool targeted by these treatments. Comparable in size to both Shire and Genzyme, Biogen Idec produces Avonex, the leading Multiple Sclerosis drug in the U.S. Should Genzyme’s Alemtuzumab MS treatment come to market, Avonex will be its primary competition.
Shire, Biogen, and Genzyme are similar in market capitalization and revenue. Genzyme's products also compete with biotech giants Abbott Laboratories and Amgen in larger patient markets. Both Abbott Labs and Amgen have products in the renal, orthopaedic, and diagnostic sectors, and while their products are not yet in direct competition with Genzyme, their larger financial resources allow them the flexibility to quickly commercialize lucrative products.
Amicus Therapeutics, a small, privately-held biotech firm, is currently developing three drugs that would directly compete with Genzyme's main products. Amicus has products in phase II trials targeting both Fabry and Gaucher disease. Their third drug, meant to treat Pompe disease, is in pre-clinical trials. While none of its drugs are ready for the market, Amicus's focus on these three niche markets could make them a serious competitor in the future.
Many of Genzyme's products are so effective and specialized that generic drug companies are expected to stay away from Genzyme's smaller markets; additionally, Genzyme's earliest patent expirations won't occur until 2010.