FDA News Roundup: Pfizer, Eli Lilly, Vertex, And More

By Anna Rose Welch, Editorial & Community Director, Advancing RNA

What FDA decisions have you missed recently?

Exelixis-Roche Faces Skin Cancer Drug Review Extension

The FDA has decided to add three months to its review period of cobimetinib. Review of the drug, indicated for skin cancer, was extended to give Roche time to submit additional data from a phase 3 registrational study investigating cobimetinib and Zelboraf for BRAF V600 mutation-positive advanced melanoma. 

Pfizer NDA Accepted For Review

The FDA has announced its plans to review Pfizer’s New Drug Application (NDA) for once daily 11 mg. Xeljanz for rheumatoid arthritis. The modified release tablets are only indicated for patients that have not been sufficiently treated with methotrexate. Xeljanz is currently approved to be taken in 5 mg doses twice-daily, either as a monotherapy or in combination with disease-modifying anti-rheumatic drugs.

Orkambi For Cystic Fibrosis Wins Approval

Vertex Pharma’s cystic fibrosis treatment, Orkambi, was approved to treat patients age 12 and up with two copies of the F508del mutation. The treatment — a combination of lumacaftor and ivacaftor — was previously granted Breakthrough Therapy designation. In a trial enrolling 1,108 participants, patients taking two Orkambi pills saw improvements in lung function.  The drug is only cleared to be taken by patients with the F580del mutation.

Catabasis DMD Drug Put On The Fast Track

CAT-1004 for Duchenne muscular dystrophy was granted Fast Track designation. The treatment targets activated NF-kB, which causes muscle degeneration and halts muscle regeneration. The orphan drug was found to be safe and well-tolerated in Phase 1 clinical trials.

Novartis Heart Failure Drug Given Approval Nod

Heart failure drug Entresto (sacubitril/valsartan) was approved under the priority review program. The drug, the first of the Angiotensin Receptor Neprilysin Inhibitors (ARNIs) family, was studied in more than 8,000 adults and proved its ability to reduce heart-failure-associated cardiovascular death and hospitalization rates when compared to competitor enalapril. Patients in the study were taking Entresto in addition to another type of treatment, including beta-blockers, diuretics, and mineralocorticoid antagonists.  The drug is expected to bring in roughly $5 billion as a treatment for the reduced ejection fraction form of heart failure.  

Dyax Wins Breakthrough Therapy Designation

DX-2930 was awarded breakthrough designation for hereditary angioedema. The subcutaneous injection, currently in the midst of a Phase 1b clinical trial, is a plasma kallikrein (pKal) inhibitor designed to prevent hereditary angioedema attacks. Compared to placebo in a proof-of-concept trial, the drug was more effective in reducing attack rates.

Summit Therapeutics Granted Fast Track Designation

SMT19969, an antibiotic for CDI, was fast tracked. Currently in Phase 2 proof-of-concept trials in North America, the drug is being developed to treat the infection following onset and to prevent recurrence.

FDA Advisory Committee Approves Of Lilly Cancer Drug

The controversial lung cancer treatment necitumumab got an informal nod of approval from an FDA advisory committee. While many have been concerned about an increased risk of fatal blood clots and electrolyte imbalances, the committee expects the drug’s benefits outshine these risks. Stage 4 non-small cell lung cancer patients in clinical trials gained 1.6 months of life by taking necitumumab in combination with gemcitabine and cisplatin.

Orphan Drug Designations

Akcea Therapeutics’ volanesorsen was named an orphan drug for Familial Chylomicronemia Syndrome (FCS). Patients taking volanesorsen in a Phase 2 study saw apoC-III triglycerides, chylomicrons, and apoC-III-associated very low density lipoprotein-cholesterol particles reduced. The drug is currently being investigated in a Phase 3 study.

Blaze Bioscience’s BLZ-100 was granted orphan designation for malignant brain tumors. A Phase 1 proof-of-concept trial is ongoing and currently enrolling adult and pediatric brain cancer patients. The drug, part of the company’s Tumor Paint platform, consists of an optimized peptide and fluorescent dye to help surgeons resecting cancer see the tumor more easily.