Progenics Pharmaceuticals Inc.'s newly approved Azedra (iobenguane I 131) arrives on the market with a nod for ultra-orphan neuroendocrine tumors, a hefty price tag and a label the New York-based firm hopes will lay the foundation for its broader radiopharmaceutical efforts.

Hoping to repeat its phase II success, Aldeyra Therapeutics Inc. enrolled the first patient in its pivotal phase III trial testing a topical version of its aldehyde trap, reproxalap, in ichthyosis associated with Sjögren-Larsson syndrome (SLS), a rare disease for which there are no approved therapies.

For Dermira Inc., winning the FDA's nod for Qbrexza (glycopyrronium cloth) as the first once-daily, topical treatment for primary axillary hyperhidrosis is only step one in its commercialization challenge. The Menlo Park, Calif.-based dermatology-focused firm now is tasked with reaching the nearly 10 million people estimated to suffer from the condition, more commonly known as excessive underarm sweating, many of whom have given up on ever finding a treatment.

As widely expected, Array Biopharma Inc.'s combo regimen of an oral, small-molecule BRAF kinase inhibitor and an oral, small-molecule MEK inhibitor cleared the FDA for use in patients with unresectable or metastatic melanoma with BRAFV600E or BRAFV600K mutations, a first approval that could prove only the tip of the iceberg for the Boulder, Colo.-based firm's development of two- and three-drug combos to follow.

Is a fourth complete response letter (CRL) in the works for embattled pain drug Remoxy ER? Wall Street seemed to think so, sending shares of developer Pain Therapeutics Inc. (NASDAQ:PTIE) down 71.5 percent to close Wednesday at $2.44, the day after a joint meeting of the FDA's Anesthetic and Analgesic Drug Products Advisory Committee and Drug Safety and Risk Management Advisory Committee voted 14-3, recommending against approval of the abuse-deterrent, extended-release, oral formulation of opioid drug oxycodone.

Barely a week after Wall Street hailed interim data from a RNA-targeting small-molecule candidate in spinal muscular atrophy (SMA) candidate, startup Skyhawk Therapeutics Inc., which also is taking a small-molecule approach to correct RNA mutations, disclosed a $40 million equity investment round and a neurology-focused option deal with Celgene Corp. that brings $60 million up front.

With one biopharma set to price its IPO this week, three others – neurology-focused Aptinyx Inc., stem cell gene therapy firm Avrobio Inc. and specialty pharma Xeris Pharmaceuticals Inc. – have set terms for their anticipated offerings, putting the sector on track to match last year's IPO activity, at least in terms of number of offerings. In terms of money raised, however, the first half of 2018 looks like it might top the amount raised in global IPOs priced in the first half of 2017.

BOSTON – When Novartis AG's Kymriah (tisagenlecleucel) became the first CAR T therapy on the market after winning FDA approval last year for B-cell precursor acute lymphoblastic leukemia (ALL), the $475,000 price tag was mitigated somewhat by news that the Swiss pharma offered an outcome-based plan in which treatment would be reimbursed only when patients responded to therapy at the end of the first month. While the move ramped up discussions on how to shift the U.S. health care economics from volume-based pricing to a value-based or outcomes-based model – a change that will become increasingly necessary as more life-changing and potentially curative gene and cell therapies reach market – cost alone marks only one of the challenges in getting CAR T treatments to patients. 

As Zymeworks Inc. prepares to unveil new phase I data from its lead bispecific antibody candidate at the upcoming American Society of Clinical Oncology meeting, its platform technology scored another win in the validation column. Less than two years after licensing rights to Zymeworks' Azymetric and EFECT technology platforms to develop one bispecific candidate, Daiichi Sankyo Co. Ltd. returned to the negotiating table for more.

Despite the rise of biologics – and biosimilars – in the ulcerative colitis (UC) space, investors backed Dublin-based Sublimity Therapeutics Ltd. in a $64 million round to advance STI-0529, its orally delivered soluble version of cyclosporine, into a phase IIb study.