Motley Fool's Top 12 Breakthroughs in Healthcare for 2016: A Retrospective on Transformative Drugs and Medical Devices

As 2016 draws to a close, let us review the achievements made in the healthcare industry this year. The U.S. Food and Drug Administration (FDA) approved 19 new drugs in 2016, covering a range of conditions from psoriasis and hepatitis C to cancer. Meanwhile, regulators also gave the green light to 25 novel medical devices in 2016. With hundreds of billions of dollars invested globally in research and development each year, it is reassuring to see that these investments have yielded drugs and devices capable of improving our health and well-being.

Of course, there are also some eye-catching breakthrough medical advancements, some of which have already been approved while others are undergoing clinical trials. For this reason, it is the renowned multimedia financial services institutionMotleyFoolA Review of the 12 Most Notable Breakthroughs in Healthcare in 2016. VCBeat (WeChat: vcbeat) has provided a detailed summary for you.

One of the most exciting developments in 2016 was the U.S. Food and Drug Administration’s (FDA) approval in September of Medtronic’s MiniMed 670G artificial pancreas, the world’s first artificial pancreas for patients with type 1 diabetes. The device, approved by the FDA for use in patients aged 14 years and older, measures blood glucose levels every five minutes. It employs a sensor with a protruding needle that penetrates beneath the skin to monitor glucose levels, while the patient wears an insulin pump on the abdomen that delivers appropriate amounts of insulin as needed.

Artificial devices can significantly reduce the incidence of hypoglycemia in patients with diabetes, eliminating the need for continuous 24-hour blood glucose monitoring. This effectively improves the quality of life and provides greater convenience for individuals with type 1 diabetes.

Furthermore, September proved to be a fortunate month for patients with type 1 diabetes, as Lexicon Pharmaceuticals (NASDAQ: LXRX) announced that its experimental dual SGLT-1 and SGLT-2 inhibitor had met the primary endpoint in a Phase 3 clinical trial for type 1 diabetes. The drug, sotagliflozin, demonstrated mean reductions in HbA1c of 0.43% and 0.49% at once-daily doses of 200 mg and 400 mg, respectively. In contrast, the placebo group showed an HbA1c reduction of only 0.08%. As the first dual inhibitor targeting SGLT-1 (which acts in the intestine) and SGLT-2 (which acts in the kidney), sotagliflozin also shares the beneficial side effects of SGLT-2 inhibitors, including weight loss and blood pressure reduction.

When it comes to the hepatitis C virus (HCV), Gilead Sciences (NASDAQ: GILD) has become synonymous with “innovation.” In June, Gilead secured approval for Epclusa, adding a third distinctive HCV medication to its portfolio. However, Epclusa is not a typical genotype-specific drug in the conventional sense; it is the first approved pan-genotypic agent. As the name suggests, “pan-genotypic” indicates its ability to treat all six genotypes of hepatitis C. For patients without cirrhosis or with mild cirrhosis, 95% to 99% achieve viral clearance 12 weeks after completing treatment. For patients with moderate to severe cirrhosis, the combination of Epclusa and ribavirin yields a sustained virologic response rate of 94% at 12 weeks post-treatment.

Duchenne Muscular Dystrophy (DMD) is an extremely rare disorder characterized by progressive skeletal muscle degeneration and atrophy due to the absence of dystrophin. In 2016, there was positive news regarding its treatment. In September, the FDA approved Sarepta Therapeutics’ (NASDAQ: SRPT) Exondys 51 therapy. This treatment is specifically designed for DMD patients amenable to exon 51 skipping, a mutation affecting approximately one in eight individuals with DMD. In multiple extension studies derived from its Phase 2b trial, Exondys 51 demonstrated significant improvement in the 6-minute walk test compared with placebo, the first FDA-approved drug for DMD.

When cannabis is mentioned, most people immediately think of drug addicts and can hardly imagine its medical value. Believe it or not, cannabis has indeed been approved for medical research. In April, the U.S. Drug Enforcement Administration (DEA) finalized the first clinical trial in which patients smoked cannabis. Although the DEA rejected a petition in August to reschedule cannabis, it gave the green light in April to a multidisciplinary association’s psychedelic research study currently underway, which aims to determine whether smoking cannabis can provide therapeutic benefits for patients with post-traumatic stress disorder (PTSD). Previous clinical studies had only permitted testing using extracts derived from the cannabis plant. Since cannabis has already shown positive effects on many diseases in earlier clinical trials, the DEA’s concession may signal that this research will advance beyond its current level.

Although Bristol-Myers Squibb’s (NASDAQ: BMY) cancer immunotherapy Opdivo has been approved for the treatment of advanced melanoma, this finding stems from a five-year follow-up study conducted after the initial Phase I trial. At the annual meeting of the American Association for Cancer Research, Bristol-Myers Squibb announced that 34% of patients who participated in the Phase I clinical trial were still alive after five years. While this figure may appear modest at first glance, it is noteworthy because prior to the introduction of cancer immunotherapy, the median survival time for patients with advanced melanoma was only 11 months.

Opdivo has long-term data for advanced melanoma, but Bristol-Myers Squibb’s key anticancer drug failed in first-line advanced non-small cell lung cancer (NSCLC) studies. However, this was not the case with its competitor, the cancer immunotherapy Keytruda. Developed by Merck & Co., Keytruda is the first PD-1 immune checkpoint inhibitor approved by the U.S. FDA. As a new class of oncology therapies, Keytruda has demonstrated significant efficacy in treating various advanced cancers, including lung cancer, renal cell carcinoma, melanoma, head and neck cancer, bladder cancer, breast cancer, liver cancer, gastric cancer, esophageal cancer, glioma, colorectal cancer, and Hodgkin lymphoma, holding promise for substantially improving patient survival.

In patients with previously untreated advanced NSCLC whose tumors express PD-L1 in at least 50% of cells, Keytruda reduced the risk of disease progression or death by 50% and the risk of death by 40% compared with chemotherapy. Furthermore, the overall response rate increased from 28% with chemotherapy to 45%. For patients with high PD-L1 expression, Keytruda has become a new hope.

Keytruda also demonstrated its first positive results in clinical trials involving patients with Merkel cell carcinoma (MCC). MCC is a rare form of skin cancer with a mortality rate approximately three times that of melanoma. In a study of 26 patients, Keytruda achieved an overall response rate of 56%.

It is worth noting the duration of response. Currently, there are no FDA-approved treatments for Merkel cell carcinoma (MCC), so the disease is typically treated with chemotherapy. Although chemotherapy yields a response rate of approximately 55%, most patients experience relapse within three months. In contrast, Keytruda does not exhibit these limitations and demonstrates durable efficacy in many patients.

Although acute migraine is difficult to treat, lasmiditan from CoLucid Pharmaceuticals (NASDAQ: CLCD) demonstrated remarkable results in September through its late-stage SAMURAI trial. Lasmiditan is a novel non-vasoconstrictive medication designed to provide treatment for migraines that have proven refractory to existing therapies.

It is a so-called “ditan” (a novel class of centrally penetrating agents) that selectively targets the trigeminal nerve via the 5-HT1F receptor. In the SAMURAI trial, 28.2% and 32.2% of patients receiving 100 mg and 200 mg doses, respectively, were free from migraine two hours after dosing, roughly double the rate observed with placebo (15.3%). Lasmiditan not only met the primary endpoint of this trial but also achieved the secondary endpoints, with 40.9% and 40.7% of patients in the 100 mg and 200 mg groups, respectively, meeting the criteria, compared with 29.5% for placebo. Assuming favorable outcomes in the remaining late-stage studies, CoLucid’s lasmiditan could become a key medication for migraine.

Treatment for patients with advanced renal cell carcinoma (RCC) has achieved new advances based on previous treatment regimens. As of April 2016, a new and effective treatment option emerged: Cabometyx from Exelixis (NASDAQ: EXEL). In the METEOR study, the primary endpoint was progression-free survival (PFS). Among the 375 patients enrolled, the median PFS was 7.4 months for cabozantinib versus 3.8 months for everolimus [HR 0.58 (95% CI: 0.45, 0.74); p < 0.0001]. The median overall survival (OS) was 21.4 months for cabozantinib versus 16.5 months for everolimus [HR 0.66 (95% CI: 0.53, 0.83); p = 0.0003]. The confirmed response rates were 17% for cabozantinib (95% CI: 2, 6) and 3% for everolimus (95% CI: 2, 6). Encouraging data from the Phase II CABOSUN study may enable Cabometyx to improve the quality of life for kidney cancer patients earlier and more rapidly.

Psoriatic arthritis is also a chronic immune-mediated inflammatory disease, characterized primarily by joint inflammation and skin lesions associated with psoriasis. Guselkumab, an experimental psoriasis drug from Johnson & Johnson (NYSE: JNJ), has also brought hope to patients by outperforming placebo in the Phase 3 VOYAGE 1 study. Guselkumab is a human monoclonal antibody that targets interleukin (IL)-23, which plays a key role in the development of immune-mediated inflammatory diseases.

Psoriasis is a chronic autoimmune inflammatory disease caused by the overproduction of skin cells, characterized by scaly, raised red inflammatory lesions or plaques that can cause itching, discomfort, and pain. It is estimated that up to 125 million people worldwide suffer from psoriasis, including more than 7.5 million patients in the United States with varying degrees of severity. Nearly one-quarter of affected individuals have moderate-to-severe disease.

Compared with only 6.9% of patients in the placebo group, 85.1% of patients receiving subcutaneous IL-23-targeted therapy achieved clear or almost clear skin after 16 weeks. Furthermore, 73.3% of patients treated with guselkumab achieved at least 90% skin clearance, compared with only 2.9% in the placebo group. At 24 weeks, 80.2% of patients in the guselkumab group achieved at least 90% skin clearance, compared with only 53% in the Humira arm, currently the world’s best-selling psoriasis medication. It goes without saying that guselkumab has the potential to significantly improve the lives of patients with psoriasis.

In May, the FDA approved a buprenorphine implant for the treatment of opioid dependence, known as Probuphine. Buprenorphine, a partial opioid receptor agonist, can also be used for maintenance therapy in addiction treatment. Developed by Titan Pharmaceuticals and Braeburn Pharmaceuticals, this implant is designed to provide patients who are stable on low-dose buprenorphine with continuous, low-dose delivery for up to six months. This new implant offers significant convenience by eliminating the need for daily pill adherence, marking another step toward safely ending the opioid epidemic in the United States.