Persistence pays off as Erbitux finally wins approval
pharmafile | February 23, 2004 | News story | |Â Â Â
Erbitux, the colorectal cancer drug once blighted by one of the biggest ever regulatory scandals has been granted marketing approval by the FDA.
In late 2001, US biotech company ImClone and marketing partner Bristol-Myers Squibb (BMS) submitted key data from clinical trials to the FDA, but saw its application rejected because of serious irregularities in the data.
The trial was intended to show Erbitux's efficacy as a combination treatment in patients with metastatic colorectal cancer who had failed to respond to irinotecan (Aventis' Campto) alone. But the regulators found a significant proportion had in fact responded to the first line treatment, with crucial safety and efficacy data also missing from half the patient records.
The plot thickened when ImClone chief executive Sam Waksal was charged with insider dealing, was subsequently found guilty and sentenced to a seven-year prison sentence.
But persistence has paid off for the companies and German pharma company Merck KGaA, which has marketing rights for the drug in Europe, where Switzerland became the first country to see its launch at the beginning of the year. Approval is expected across the European Union by mid-2004, and Merck is confident that research conducted in 11 European countries contains none of the irregularities seen in the early US trials.
Sam Waksal's brother Harlan succeeded him as chief executive of ImClone for a brief period, but he too was forced to stand down last April because of a federal tax and accounting investigation.
Harlan Waksal was never charged with any wrongdoing, and issued a personal statement on the news of the FDA approval, saying it was a "major personal milestone for me and my family".
"I am especially pleased for the tens of thousands of cancer patients who will have access to this new drug as part of their treatment plan," he added. "I am grateful to all of the employees of ImClone for their perseverance through difficult times in bringing this drug to market. This approval is a vindication for those who always believed in the promise and prospects for Erbitux."
Erbitux (cetuximab) is one of a class of new cancer drugs which target genetic mutations which allow cancer cells to multiply, and is designed to bypass many of the unpleasant side effects associated with traditional chemotherapy.
Other drugs that work on the same principle of blocking the epidermal growth factor receptor (EGFR) include AstraZeneca's Iressa (gefitinib) and Roche's Tarceva (erlotinib).
Erbitux will compete with these drugs to treat other cancers where EGFR is involved, such as head and neck cancers and other solid tumours.
The drug will face competition in the colorectal cancer drug market from Genentech's Avastin, which was filed in the US in September and three months later in the EU and is expected to gain approval in both markets sometime this year.
Avastin is the first proven cancer drug to target another tumour growth-promoting protein, vascular endothelial growth factor (VEGF), and will be used as a first line treatment for patients with advanced colorectal cancer.
Analysts differ on how successful Erbitux can be in the markets, but Standard & Poor's say US sales could reach a peak of $1.5 billion in 2012, and top EU sales of $600million in the same period.
The drug's US approval is vital for BMS as well as ImClone: the former continues to suffer from patent losses to its flagship oncology franchise, with Taxol to follow in some EU countries this year.
The tale of Erbitux has also been seen as a case study on in-licensing, with ImClone successfully resisting pressure from its larger partner's attempts to re-negotiate the terms of their deal on royalties and profit-sharing.
