-----BEGIN PRIVACY-ENHANCED MESSAGE----- Proc-Type: 2001,MIC-CLEAR Originator-Name: webmaster@www.sec.gov Originator-Key-Asymmetric: MFgwCgYEVQgBAQICAf8DSgAwRwJAW2sNKK9AVtBzYZmr6aGjlWyK3XmZv3dTINen TWSM7vrzLADbmYQaionwg5sDW3P6oaM5D3tdezXMm7z1T+B+twIDAQAB MIC-Info: RSA-MD5,RSA, TOUqiAo2P8q6/J/CNAHucVg/QPUkdGgJpT8PLytWCalGJVA/3SJlFziJyoxLIdE4 9foNwRH2Tl9zmyuC5/X++Q== 0001048477-02-000075.txt : 20020917 0001048477-02-000075.hdr.sgml : 20020917 20020916194353 ACCESSION NUMBER: 0001048477-02-000075 CONFORMED SUBMISSION TYPE: 8-K PUBLIC DOCUMENT COUNT: 2 CONFORMED PERIOD OF REPORT: 20020916 ITEM INFORMATION: Other events FILED AS OF DATE: 20020917 FILER: COMPANY DATA: COMPANY CONFORMED NAME: BIOMARIN PHARMACEUTICAL INC CENTRAL INDEX KEY: 0001048477 STANDARD INDUSTRIAL CLASSIFICATION: PHARMACEUTICAL PREPARATIONS [2834] IRS NUMBER: 680397820 STATE OF INCORPORATION: DE FISCAL YEAR END: 1231 FILING VALUES: FORM TYPE: 8-K SEC ACT: 1934 Act SEC FILE NUMBER: 000-26727 FILM NUMBER: 02765380 BUSINESS ADDRESS: STREET 1: 371 BEL MARIN KEYS BLVD STREET 2: STE 210 CITY: NOVATO STATE: CA ZIP: 94949 BUSINESS PHONE: 4158846700 MAIL ADDRESS: STREET 1: 371 BEL MARIN KEYS BLVD STREET 2: STE 210 CITY: NOVATO STATE: CA ZIP: 94949 8-K 1 bmrn8k091602.txt FORM 8-K PRESS RELEASE ================================================================================ SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 8-K CURRENT REPORT Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934 Date of Report (Date of earliest event reported): September 16, 2002 BioMarin Pharmaceutical Inc. (Exact name of registrant as specified in its charter) Delaware 000-26727 68-0397820 (State or other jurisdiction of (Commission (IRS Employer incorporation or organization) File Number) Identification No.) 371 Bel Marin Keys Boulevard, Suite 210, Novato, California 94949 (Address of principal executive offices) (Zip Code) Registrant's telephone number, including area code: (415) 884-6700 Not Applicable ------------------------------------------------------------- (Former name or former address, if changed since last report) ================================================================================ ================================================================================ Item 5. Other Events. On September 16, 2002, BioMarin Pharmaceutical Inc. (the "Registrant"), together with its joint venture partner Genzyme General, announced the U.S. Food and Drug Administration has accepted for review the Biologics License Application (BLA) filing for Aldurazyme, and has granted the application priority review status. The Registrant's press release issued on September 16, 2002 is attached hereto as Exhibit 99.1. Item 7. Financial Statements, Pro Forma Financial Statements and Exhibits. (a) Financial Statements of Business Acquired. Not Applicable. (b) Pro Forma Financial Information. Not Applicable. (c) Exhibits. Exhibit 99.1 Press Release of the Registrant dated September 16, 2002. SIGNATURE Pursuant to the requirements of the Securities Exchange Act of 1934, the Registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized. BioMarin Pharmaceutical Inc., a Delaware corporation Date: September 16, 2002 By: /s/ Fredric D. Price -------------------- Fredric D. Price Chairman and Chief Executive Officer EXHIBIT INDEX Exhibit No. Description - ----------- ----------- Exhibit 99.1 Press Release of the Registrant dated September 16, 2002. EX-99 3 bmrn8kfdapriorityreview.txt FORM 8-K PRESS RELEASE Exhibit 99.1 CONTACTS: For BioMarin For Genzyme - ------------ ----------- Joshua Grass (Investors) Sally Curley (Investors) (415) 884-6777 (617) 591-7140 Fredda Malkoff (Media) Dan Quinn (Media) Feinstein Kean Healthcare (617) 591-5849 (617) 577-8110 For Immediate Release BioMarin and Genzyme's Application for Aldurazyme Accepted by the FDA and Granted Priority Review Status Novato, CA and Cambridge, MA, September 16, 2002 - BioMarin Pharmaceutical Inc. (Nasdaq and Swiss SWX New Market: BMRN) and Genzyme General (Nasdaq: GENZ) today announced that the U.S. Food and Drug Administration (FDA) has accepted for review their Biologics License Application (BLA) filing for Aldurazyme(TM), and has granted the application priority review status. Priority review status specifies that the FDA will respond to the filing within six months from the date of the completed BLA filing, which was July 29, 2002. Priority review is an FDA procedure designed to accelerate the review of products that address an unmet medical need. Aldurazyme is an investigational enzyme replacement therapy for patients with mucopolysaccharidosis I (MPS I), a progressive, debilitating and fatal genetic disease for which no specific drug treatments currently exist. BioMarin and Genzyme included clinical data from the six-month, placebo-controlled Phase 3 trial of Aldurazyme, six months of data from the ongoing open-label Phase 3 extension study, and three years of data from the Phase 1 trial and extension study. On March 1, 2002, BioMarin and Genzyme also submitted a Marketing Authorization Application (MAA) to the European Agency for the Evaluation of Medicinal Products (EMEA) for approval to market Aldurazyme in the European Union. The Agency accepted the MAA and is currently reviewing the application. BioMarin and Genzyme expect a response from the EMEA in the first half of 2003. About MPS I - ----------- MPS I is a progressive, debilitating and fatal genetic disease caused by a deficiency of the enzyme alpha-L-iduronidase. This deficiency leads to the accumulation of complex carbohydrates in the lysosomes of cells, leading to the progressive dysfunction of cellular, tissue and organ systems. Resulting symptoms, which span a spectrum of severity, can include impaired cardiac and pulmonary function, delayed physical development, skeletal and joint deformities, reduced endurance, and in some cases, delayed mental function. A majority of patients die before adulthood from complications of the disease. BioMarin and Genzyme formed a joint venture in 1998 to develop and commercialize Aldurazyme worldwide. Under the terms of the joint venture, if approved for commercial sale, BioMarin will manufacture Aldurazyme and Genzyme will have responsibility for the commercialization of the product. The companies have obtained Orphan Drug designation and Fast Track status for Aldurazyme for the treatment of MPS I from the FDA and orphan medicinal product designation from the EMEA. Genzyme General develops and markets therapeutic products and diagnostic products and services. Genzyme General has five therapeutic products on the market and a strong pipeline of therapeutic products in development focused on the treatment of genetic diseases and other chronic debilitating disorders with well-defined patient populations. Genzyme General is a division of Genzyme Corp. BioMarin Pharmaceutical specializes in the development and commercialization of therapeutic enzyme products to treat serious, life-threatening diseases and conditions. This press release contains forward-looking statements, including without limitation statements about: decisions by the FDA and the EMEA and the expected timing thereof; the potential approval of Aldurazyme for commercial sale; and commercialization plans. These statements are subject to risks and uncertainties that could cause actual results to differ materially from those projected in these forward-looking statements. These risks and uncertainties include, among others: the content and timing of decisions made by the FDA and the EMEA regarding regulatory submissions for Aldurazyme; the ability to manufacture sufficient quantities of product for commercialization activities and to do so in a timely manner; the continued funding of the joint venture between Genzyme and BioMarin; decisions made by physicians and third party payers regarding the use of and reimbursement for Aldurazyme; our ability to obtain and maintain adequate patent and other proprietary rights protection for Aldurazyme; the scope, validity and enforceability of patents and other proprietary rights held by third parties related to therapies for MPS I and the actual impact of such patents and other rights on our ability to commercialize Aldurazyme; the competitive environment for therapies for MPS I; and the risks and uncertainties described in reports filed by Genzyme and BioMarin with the Securities and Exchange Commission under the Securities Exchange Act of 1934, as amended, including without limitation the factors contained under the caption "Factors Affecting Future Operating Results" in Exhibit 99.2 to Genzyme Corporation's 2001 Annual Report on Form 10-K. Genzyme General Division common stock is a series of common stock of Genzyme Corporation. Therefore, holders of Genzyme General Division common stock are subject to all of the risks and uncertainties described in the those reports. Genzyme and BioMarin caution investors not to place undue reliance on the forward-looking statements contained in this press release. These statements speak only as of the date of this press release, and Genzyme and BioMarin undertake no obligation to update or revise the statements. Genzyme(R) is a registered trademark of Genzyme Corporation. AldurazymeTM is a trademark of BioMarin/Genzyme LLC. All rights reserved. # # # Genzyme's press releases and other company information are available at www.genzyme.com and by calling Genzyme's investor information line at 1-800-905-4369 within the United States or 1-703-797-1866 outside the United States. BioMarin's press releases and other company information are available online at http://www.biomarinpharm.com. -----END PRIVACY-ENHANCED MESSAGE-----