Novartis wins a record breakthrough therapy designation for orphan drug

Novartis picked up its third breakthrough therapy designation from the FDA, a coveted record number of programs in the Big Pharma crowd.

The agency tapped BYM338 (bimagrumab), an antibody developed in collaboration with MorphoSys for a rare and potentially lethal muscle-wasting disease called sporadic inclusion body myositis. According to Novartis ($NVS), the agency issued the breakthrough designation after reviewing promising Phase II data, now slated for public release on October 14.

BYM338--an IV treatment--latches to type II activin receptors, blocking natural ligands like myostatin and activin, spurring muscle development by shutting down the signals from these inhibitory molecules. And in addition to sIBM, the therapy is also being developed for COPD, cancer cachexia, sarcopenia and mechanically ventilated patients.

Pharmacyclics ($PCYC) has received three breakthrough designations, but all for the same drug--ibrutinib. The company has credited the designation with chopping 7 to 9 months off of the regulatory process by allowing the biotech, partnered with J&J ($JNJ), to use Phase II data for the initial cancer application. And the agency is promising to potentially slice years out of the development timelines for breakthrough drugs, which are largely controlled by Big Pharma companies.

Novartis has also won breakthrough designations for the cancer drug LDK378 and serelaxin for acute heart failure. You can find a list of the publicly announced breakthrough drugs at the Orphan Druganaut blog.

The news about the breakthrough designation may help reawaken periodic rumors of a Novartis buyout of MorphoSys. The two companies recently expanded a long-standing development pact and some European analysts have been considering a possible M&A deal after Joerg Reinhardt, a former member of the MorphoSys supervisory board, came on as chairman of the pharma giant.

"BYM338 is the third example this year of Novartis' leadership in bringing breakthrough therapies to patients reinforcing our commitment to innovation addressing significant unmet medical needs and enhancing the lives of patients," said Timothy Wright, the global head of development for Novartis. "With no effective therapies currently available for sIBM, bimagrumab has the potential to be the first real option for patients with this condition."

For the record, J&J's collaboration with Pharmacyclics along with its breakthrough designation for daratumumab gives it four breakthrough designations on two drugs.

- here's the press release

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