Regulatory Roundup: Top News Stories – Week Ending July 14, 2017

Lots of regulatory news this week including one new molecular entity approval, an Advisory Committee thumbs-up for Novartis’s CAR-T program, a reversal for Amicus, and several new orphan drug designations. Here’s my summary of the top regulatory stories for this week:

J&J subsidiary Janssen Biotech has secured approval from the FDA for its Tremfya (guselkumab) to treat adults with moderate to severe plaque psoriasis. This is the 24th new molecular entity to be approved by the FDA in 2017

The FDA granted approval to Emmaus Medical’s Endari (L-glutamine oral powder), the first treatment approved for patients with sickle cell disease in almost 20 years.

The FDA’s advisory committee on cancer drugs (ODAC) voted unanimously in favor of Novartis’ trailblazing CAR-T CTL019 (tisagenlecleucel), marking a key milestone for the pharma giant as it nears the likely launch of a groundbreaking personalized cell therapy for B-cell acute lymphoblastic leukemia.

ODAC also unanimously backed the approval of Amgen and Mylan’s biosimilar versions of two of Roche’s top selling cancer drugs, Avastin (bevacizumab) and Herceptin (trastuzumab).

The FDA Oncologic Drug Advisory Committee (ODAC) voted in Favor of Pfizer’s MYLOTARG (gemtuzumab ozogamicin) for Acute Myeloid Leukemia

The EMA announced that it is provisionally restricting the use of Biogen’s multiple sclerosis medicine Zinbryta (daclizumab) to patients with highly active relapsing disease that have failed to respond to other drugs, and to patients with rapidly evolving relapsing disease who cannot be treated with other medicines.

Concert Pharmaceuticals announced that the FDA has lifted the clinical hold on Concert’s CTP-543 Phase 2a clinical trial for alopecia areata. Concert will resume enrollment in the Phase 2a trial later this month and expects to complete the trial in the second half of 2018.

Eli Lilly announced that the FDA has accepted its NDA for abemaciclib (a cyclin-dependent kinase (CDK)4 & 6 inhibitor for the treatment of breast cancer) and given the NDA a Priority Review designation.

Ignyta, Inc. announced that the FDA has granted orphan drug designation to entrectinib for treatment of NTRK fusion-positive solid tumors.

Boston Biomedical announced that the FDA has granted Orphan Drug Designation for DSP-7888, an investigational cancer peptide vaccine, for the treatment of myelodysplastic syndrome.

Reata Pharmaceuticals announced that the FDA has granted orphan designation to bardoxolone methyl for the treatment of Alport syndrome, a rare genetic form of chronic kidney disease.

KaloBios received Orphan Drug Designation for Benznidazole for the treatment of Chagas Disease. KaloBios expects to submit a New Drug Application (NDA) submission for benznidazole in the first quarter of 2018.

Principia Biopharma announced that PRN1008, a reversible covalent Bruton’s Tyrosine Kinase inhibitor, has been granted orphan drug designation by the FDA for the treatment of patients with pemphigus vulgaris.

The FDA granted orphan drug designation to Mallinckrodt’s MNK-1411 for potential treatment of Duchenne Muscular Dystrophy.

Allena Pharmaceuticals announced that the FDA has granted orphan drug designation to Allena’s investigational product ALLN-177, an oral formulation of oxalate decarboxylase, for the treatment of primary hyperoxaluria.

The FDA has accepted for priority review a sNDA submitted by Bristol-Myers Squibb for Sprycel (dasatinib) to treat children with Philadelphia chromosome-positive chronic phase chronic myeloid leukemia (CML).

Amicus Therapeutics announced on Tuesday morning that the FDA had given it the all-clear to submit for review its treatment for Fabry disease. Just last year, the FDA asked Amicus to do another study on side effects, which would have delayed approval by two years. The unusual move comes after President Trump met with the company’s CEO — and promised to speed up what he called a “slow and burdensome” process for drug approvals.

The FDA rejected Ocular Therapeutix eye drug Dextenza a second time, citing manufacturing issues.

In other news…

In a setback for Gilead Sciences, a federal appeals court reinstated a whistleblower lawsuit filed by two former employees, who accused the company of misleading regulators about contaminated ingredients used in various HIV medicines and falsifying data to win marketing approval for the drugs.

Emergent BioSolutions signed an agreement with Sanofi Pasteur to acquire their ACAM2000 smallpox vaccine business.

As part of an effort to expand its flu-vaccine portfolio, Sanofi will acquire biotech firm Protein Sciences for $650 million.

More than a dozen drug developers went public between April 1 and June 30 of this year. The list includes Tocagen, Verona Pharma, Zymeworks, UroGen Pharma, Biohaven Pharmaceuticals, Ovid Therapeutics, G1 Therapeutics, Argenx, Athenex, Avenue Therapeutics, Mersana Therapeutics, Dova Pharmaceuticals and Aileron Therapeutics.

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