Genmab A/S's HuMax-CD4 Receives EU Orphan Drug Designation
COPENHAGEN, Denmark, May 25 /PRNewswire-FirstCall/ -- Genmab A/S announced today that HuMax-CD4 for the treatment of Cutaneous T-cell Lymphoma (CTCL) has been designated an orphan drug by the European Agency for the Evaluation of Medicinal Products (EMEA).
The filing for this status was completed as part of an overall plan to obtain orphan drug protection for HuMax-CD4 to treat CTCL. At present Genmab's primary rights for HuMax-CD4 are in North and South America, and Genmab does not hold rights to HuMax-CD4 in Europe.
Under the EMEA's Regulation (EC) No. 141/2000 an orphan drug designation gives companies access to protocol assistance and guidance on preparing a dossier that will meet European regulatory requirements and thereby maximize the chance of success at the time of marketing authorization. Once approved, an orphan drug is also granted 10 years of market exclusivity during which directly competitive similar products cannot normally be placed on the market.
The EMEA grants orphan drug designation based upon several criteria: the life-threatening and debilitating nature of the condition; the medical plausibility of the proposed orphan indication; a prevalence in Europe of less than 5 cases for each 10,000 of population; no satisfactory method of diagnosis, prevention or treatment exists or if such method exists the medicinal product will be of significant benefit to those affected by that condition.
"The EMEA has granted orphan drug status to HuMax-CD4 on the basis of the available safety and efficacy data which shows the antibody may be of significant benefit to CTCL patients," said Lisa N. Drakeman, Ph.D., Chief Executive Officer of Genmab. "The orphan designation strengthens our chances of bringing HuMax-CD4 to patients who desperately need alternative methods of treatment."
HuMax-CD4 is a high affinity human antibody that targets the CD4 receptor on T-lymphocytes. Genmab has run two Phase II studies using HuMax-CD4 to treat CTCL, one in early stage patients and the other for patients with advanced disease, both of which achieved positive results.
Phase II Studies Response Rate
Overall in the Phase II studies, 55% of MF patients (10/18) treated at higher doses achieved at least a partial response. Of fourteen early stage MF patients treated at the 560mg dose level, seven achieved at least a partial response, including two who obtained a complete response. Three of four advanced stage MF patients (75%) treated at the 980mg level obtained a partial response. There was also a statistically significant dose response correlation in 38 early and late stage mycosis fungoides patients.
HuMax-CD4 is considered to be safe and well-tolerated in clinical trials to date. Of the 47 patients treated in the Phase II studies at all dose levels, only five grade-three events (hypersensitivity, elevated liver enzymes, aggravated pruritus [2 patients], and muscle fiber rupture) were considered potentially related to HuMax-CD4 treatment, based on judgments by the treating physicians.
T-cell lymphomas positive for the CD4 receptor constitute around 5% of Non-Hodgkin's Lymphomas. The US prevalence of the MF is estimated at 16,000 to 20,000. CTCL patients tend to have a lifespan of 10 to 30 years and therefore could be treated several times during the disease progression.
Non-cutaneous CD4 positive T-cell lymphomas originate predominantly in the lymph nodes. Their prevalence in the US and Canada is approximately 8,000 to 10,000.
About Genmab A/S
Genmab A/S is a biotechnology company that creates and develops human antibodies for the treatment of life-threatening and debilitating diseases. Genmab has numerous products in development to treat cancer, infectious disease, rheumatoid arthritis and other inflammatory conditions, and intends to continue assembling a broad portfolio of new therapeutic products. At present, Genmab has multiple partnerships to gain access to disease targets and develop novel human antibodies including agreements with Roche and Amgen. A broad alliance provides Genmab with access to Medarex, Inc.'s array of proprietary technologies, including the UltiMAb(TM) platform for the rapid creation and development of human antibodies to virtually any disease target. Genmab is headquartered in Copenhagen, Denmark and has operations in Utrecht, The Netherlands and Princeton, New Jersey in the US. For more information about Genmab, visit http://www.genmab.com/.
Except for the historical information presented herein, matters discussed in this press release are forward-looking statements that are subject to certain risks and uncertainties that could cause actual results to differ materially from any future results, performance or achievements expressed or implied by such statements, e.g. unforeseen exchange rate and interest rate fluctuations, delayed or unsuccessful development projects.
Statements that are not historical facts, including statements preceded by, followed by, or that include the words "believes"; "anticipates"; "plans"; "expects"; "estimates"; or similar statements are forward-looking statements. Genmab is not under an obligation to update statements regarding the future following the publication of this release; nor to confirm such statements in relation to actual results, unless this is required by law.Genmab A/S
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