Insmed Incorporated Tanks As Phase 3 Plans Are Needed For ARIKAYCE, Delaying FDA Approval

Published: Aug 05, 2014

Insmed Incorporated Tanks As Phase 3 Plans Are Needed For ARIKAYCE, Delaying FDA Approval Insmed Incorporated Tanks As Phase 3 Plans Are Needed For ARIKAYCE, Delaying FDA Approval
August 5, 2014
By Jessica Wilson, Breaking News Staff

Insmed Incorporated , a biopharmaceutical company focused on lung diseases, saw its share price plummet yesterday, with a dramatic increase in the number of shares exchanged. Its stock movement came in the wake of the FDA decision that Insmed must move its drug ARIKAYCE™ to Phase III trials, which will delay FDA approval and thus the drug’s time-to-market.

In an announcement released yesterday, the company emphasizes its determination to conduct the trials in the shortest timeframe possible in order to achieve the quickest path to filing for FDA approval.

Yesterday, shares of Insmed fell by more than 25%, with the volume traded increasing from an average of 883,698 to 3.9 million shares.

While it is common for the FDA to require Phase III trials before approving a drug, there are exceptions. In a 2001 testimony in front of Congress, a representative from the FDA stated that, “In some cases the Phase II studies reveal results so impressive that these studies alone are the basis for approval, generally for treatment of refractory disease.”

A refractory disease is one resistant to existing treatment. ARIKAYCE™ is designed to treat, among other conditions, “nontuberculous mycobacteria (NTM) lung infections in treatment refractory patients,” according to Insmed. In addition, the FDA has granted ARIKAYCE™ Breakthrough Therapy, Orphan Drug, Qualified Infectious Disease Product (QIDP) and Fast Track designations. The European Medicines Agency (EMA), the drug regulatory body in Europe, has also granted ARIKAYCE™ Orphan Drug Designation. The aim of all these designations is to expedite a drug to market. Thus, the requirement of Phase III trials caused surprise among investors and led to the fall of Insmed’s share price.

ARIKAYCE™ is a form of the antibiotic amikacin designed to be a once-daily medication inhaled via the eFlow® Nebulizer System manufactured by PARI Pharma GmbH. ARIKAYCE™ is enclosed by liposomes, or nanocapsules of lipid, in order to prolong the release of the antibiotic. The aim is to treat both Nontuberculous Mycobacteria lung disease (NTM) and Pseudomonas lung infections, often acquired by individuals with Cystic Fibrosis.

Instances of NTM, for which no effective treatment exists, have increased in the US and other developed countries. NTM, a chronic condition, causes inflammation and lung damage, which lead to extended hospital stays for most patients. Approximately 30,000 cases of pulmonary disease thought to be caused by NTM lung infections exist in Europe and another 55,000 cases of NTM lung disease exist in the US.

People with Cystic Fibrosis comprise a group particularly vulnerable to lung infections difficult to treat. Worldwide, approximately 70,000 patients have CF, with 35,000 in Europe and 30,000 in the US. CF, an inherited and chronic disease, causes life-threatening lung infections by programming the body to manufacture thick mucus that clogs the lungs. More than half of patients with CF develop Pseudomonas lung infections by age 18, which, due to the effects of the disease, become impossible to clear and lead to severe decline in lung function.

Insmed has stated it will file a Marketing Authorization Application (MAA) with the European Medicines Agency by the end of 2014, which is the next step for a commercial launch in Europe. The company will run additional trials in the US; the results from the first trial are expected in 2016 and from a second larger trial in 2017.

Read more recent MAA news.
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