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Clinical Notes: European Panel OKs Gene Therapy

By John Gever, , MedPage Today

A gene therapy for an enzyme deficiency disease is just one step short of marketing approval in Europe. Also this week: the EMA nixes calcitonin drugs for long-term use.

Gene Therapy Nears European Approval

What could be the first gene therapy to win approval in the developed world received a favorable review from the European Medicines Agency’s (EMA) top advisory committee.

The treatment, called Glybera, would replace a defective gene in patients with lipoprotein lipase deficiency (LPLD).

It’s the first gene therapy to be recommended by the EMA’s Committee for Medicinal Products for Human Use (CHMP). European regulators almost always follow the panel’s recommendations.

But the committee’s vote came with two caveats. First, the approval recommendation was limited to LPLD patients with multiple or severe pancreatitis attacks. Second, the product’s sponsor, Amsterdam-based UniQure, must set up a patient registry to provide data for postmarketing surveillance.

No gene therapy has progressed this far in the U.S. Indeed, it took UniQure 3 years from its original EMA marketing application to reach this point. Twice last year, the CHMP recommended against approval in all LPLD patients. Members changed their minds only after the narrower indication was offered, the EMA said.

EMA Panel Seeks to Curb Calcitonin Drugs

Calcitonin-based drugs should not be given for long periods because of increased risk of cancer with chronic treatment, the CHMP indicated in another decision.

The panel called for outright withdrawal of intranasal formulations of calcitonin, since these are only used for osteoporosis and hence are intended for long-term administration.

Long-term trials indicated that risks of various cancers were increased by 0.7 to 2.4 percentage points in patients receiving such agents.

For indications other than osteoporosis, the CHMP voted to keep calcitonin approvals in place for injection or infusion, but with a recommendation to keep dosing to “the shortest possible time,” according to an EMA press release.

Permissible indications include Paget’s disease not responding to other drugs, cancer-related hypercalcemia, and prevention of bone loss due to immobilization.

A combined blood glucose meter and insulin pump that can be operated remotely won FDA approval, its manufacturer said.

Made by Roche, the Accu-Chek Combo system links the meter and pump with wireless Bluetooth technology that grants data to move in both directions.

Roche stated the remote control feature lets patients administer a bolus insulin dose, when the meter indicates one is called for, without touching the pump.

FDA Punts Insulin Degludec Decision

Novo Nordisk stated the FDA would not approve its ultra-long acting insulin product, insulin degludec, in late October as it had previously indicated.

Instead, the bureau has scheduled an advisory committee meeting for November 8 to review the drug and a related product that combines insulin degludec with insulin aspart.

The company did not say what concerns led the FDA to seek the outside opinion. Novo Nordisk had submitted marketing applications for the two products last September. Only last month the FDA had stated it would render a decision by October 29.

States Move to Solo Pentobarbital for Executions

Prison officials in Georgia are planning to join their counterparts in Texas in using a single drug, pentobarbital, to carry out executions.

No reason was given, but Georgia was one of several says from which federal officials had previously confiscated supplies of sodium thiopental, another common execution drug, that it stated were imported illegally.

Traditionally, lethal injections have been carried out with a cocktail of three drugs, usually including sodium thiopental, pancuronium bromide, and pentobarbital. But European manufacturers of these agents have opposed their use in executions and have implemented measures to keep them away from U.S. prison authorities.

Along with more prosaic drug shortages — the sole U.S. manufacturer of thiopental ended up quitting the business after manufacturing problems cropped up — the European efforts led some says to seek supplies from the pharmaceutical gray market.

Recently, prison officials in Texas stated they would begin using pentobarbital alone in executions and Georgia followed suit. However, the first execution in the latter say was briefly postponed while authorities finalized the new protocol, Reuters reported.

Anemia Drug Hits Phase III Endpoints

A phase III trial of ferumoxytol (Feraheme) indicated that it boosted hemoglobin counts in patients with iron deficiency anemia irrespective of cause, the drug’s manufacturer said.

The product is currently approved to treat anemia related to chronic kidney disease. Its maker, AMAG Pharmaceuticals, is hoping to expand its indications to include all patients with iron deficiency anemia regardless of the cause.

In the new 808-patient trial, 5 weeks of treatment led to increases in mean hemoglobin of 2.7 g/dL, compared with 0.1 g/dL with placebo. The comparison was one of two co-primary endpoints in the trial.

More than 80% of patients met the other primary endpoint of an increase of at least 2 g/dL versus 6% of the placebo group.

AMAG stated it planned to request approval for the expanded indication by the end of the year.

, , has covered biomedicine and medical technology for 30 years. He holds a B.S. from the University of Michigan and an M.S. from Boston University. Now based in Pittsburgh, he is the daily assignment editor for MedPage Today as well as general factotum on the reporting side. Go Pirates/Penguins/Steelers!

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